What is Cabometyx?

Cabometyx is used to treat certain types of kidney cancer (renal cell carcinoma), liver cancer (hepatocellular carcinoma), differentiated thyroid cancer, and pancreatic and extra-pancreatic neuroendocrine tumors. It is an oral tablet taken once daily.

Cabometyx (cabozantinib) gained FDA approval on November 29, 2012. There is no generic.

FDA Approvals and Uses

Cabometyx (cabozantinib) is an oral kinase inhibitor approved to treat:

• Advanced renal cell carcinoma (RCC that has spread), as monotherapy
• Advanced RCC as first-line treatment in combination with nivolumab
• Hepatocellular carcinoma in patients previously treated with sorafenib
• Locally advanced or metastatic differentiated thyroid cancer (DTC) in adults and children 12 years and older that has progressed following VEGFR-targeted therapy and who are radioactive iodine-refractory or ineligible.
• Previously treated, unresectable, locally advanced or metastatic, well-differentiated pancreatic neuroendocrine tumors (pNET) or extra-pancreatic neuroendocrine tumors (epNET) in adults and children 12 years and older

It is not known if Cabometyx is safe and effective in children younger than 12 years of age.

Side Effects

Common Side Effects

The most common side effects of Cabometyx are:

• Tiredness
• Nausea and vomiting
• Constipation
• Decreased appetite
• Weight decreased.

The most common side effects of Cabometyx when used in combination with nivolumab are:

• Tiredness
• Mouth sores
• Rash
• Low thyroid hormone levels (hypothyroidism)
• Pain in muscles, bones, and joints
• Decreased appetite
• Nausea
• Changes in the way things taste
• Stomach-area (abdominal) pain
• Cough
• Upper respiratory tract infection.

Cabometyx may cause fertility problems in females and males, which may affect your ability to have children. Talk to your healthcare provider if you have concerns about fertility.

Serious side effects and warnings

Cabometyx may cause the following serious side effects.

Bleeding (hemorrhage)

Cabometyx can cause severe bleeding that may lead to death. Tell your healthcare provider right away if you get any signs of bleeding during treatment with Cabometyx, including:

• Coughing up blood or blood clots
• Vomiting blood, or if your vomit looks like coffee grounds
• Red or black (looks like tar) stools
• Menstrual bleeding that is heavier than normal
• Any unusual or heavy bleeding

Perforations and fistulas

Cabometyx can cause a tear in your stomach or intestinal wall (perforation) or an abnormal connection between 2 parts of your body (fistula). Tell your healthcare provider right away if you get tenderness or pain in your stomacharea (abdomen) that is severe, or that does not go away.

Blood clots, stroke, heart attack, and chest pain

Cabometyx may affect the way your blood clots and increase the risk of blood clots, a stroke, heart attack, or chest pain. Get emergency help right away if you get:

• Swelling or pain in your arms or legs
• Shortness of breath
• Feel lightheaded or faint
• Sweating more than usual
• Numbness or weakness of your face, arm or leg, especially on one side of your body
• Sudden confusion, trouble speaking, or understanding
• Sudden trouble seeing in one or both eyes
• Sudden trouble walking
• Dizziness, loss of balance, or coordination
• A sudden severe headache.

High blood pressure (hypertension)

Hypertension is common with Cabometyx and sometimes can be severe. Your healthcare provider will check your blood pressure before starting Cabometyx and regularly during treatment. If needed, your healthcare provider may prescribe medicine to treat your high blood pressure. Tell your healthcare provider if you develop severe headaches, nose bleeds, tiredness or confusion, vision changes, chest pain, trouble breathing, irregular heartbeat, or blood in your urine.

Heart problems

Cabometyx can cause heart failure that may lead to death. Your healthcare provider may check your heart function before and during treatment with Cabometyx. Tell your healthcare provider right away if you get any of the following signs and symptoms:

• Feeling like your heart is pounding, racing, or beating irregularly
• Swelling of your ankles or feet
• Feeling lightheaded
• Shortness of breath
• Tiredness

Diarrhea

Diarrhea is common with Cabometyx and can be severe. If needed, your healthcare provider may prescribe medicine to treat your diarrhea. Tell your healthcare provider right away if you have frequent loose, watery bowel movements.

Hand-foot skin reaction

Hand-foot skin reactions are common with Cabometyx and can be severe. Tell your healthcare provider right away if you have rashes, redness, pain, swelling, or blisters on the palms of your hands or soles of your feet.

Liver problems

Liver problems may happen during treatment with Cabometyx. When Cabometyx is taken in combination with nivolumab, severe changes in liver function tests may happen more often than if you take Cabometyx alone. Your healthcare provider will do blood tests to check your liver function before and during treatment with Cabometyx. Tell your healthcare provider right away if you develop symptoms of liver problems, including yellowing of your skin or the whites of your eyes, severe nausea or vomiting, pain on the right side of your stomach-area (abdomen), dark urine, bleeding, or bruising more easily than normal.

Adrenal gland problems

Your healthcare provider will monitor you for this problem. Your healthcare provider may prescribe hormone replacement therapy or corticosteroid medicines if needed. Tell your healthcare provider right away if you develop any of the following signs or symptoms: extreme tiredness, dizziness or fainting, weakness, nausea, or vomiting.

Protein in your urine and possible kidney problems

Symptoms may include swelling in your hands, arms, legs, or feet. Your healthcare provider will check you for this problem during treatment with Cabometyx.  

Severe jawbone problems (osteonecrosis)

Your healthcare provider should examine your mouth before you start and during treatment with Cabometyx. Tell your dentist that you are taking Cabometyx. It is important for you to practice good mouth care during treatment with Cabometyx. Tell your healthcare provider right away if you develop any symptoms of jaw problems, including: jaw pain, toothache, or sores on your gums.

Wound healing problems

Wound healing problems have happened in people who take Cabometyx. Tell your healthcare provider if you plan to have any surgery before or during treatment with Cabometyx.

• You should stop taking Cabometyx at least 3 weeks before planned surgery.
• Your healthcare provider should tell you when you may start taking Cabometyx again after surgery.

Reversible Posterior Leukoencephalopathy Syndrome (RPLS)

A condition called reversible posterior leukoencephalopathy syndrome can happen during treatment with Cabometyx. Tell your healthcare provider right away if you have headaches, seizures, confusion, changes in vision, or problems thinking.

Change in thyroid function

Cabometyx can cause changes in your thyroid function, including changes to thyroid hormone levels in your blood. Your healthcare provider will do blood tests to check your thyroid function before and during treatment with Cabometyx.

Decreased calcium level in your blood (hypocalcemia)

Cabometyx can cause you to have a decreased amount of calcium in your blood. Your healthcare provider will do blood tests to check you for this problem and give you calcium if needed. Tell your healthcare provider right away if you get any of the following signs or symptoms:

• Muscle stiffness or muscle spasms
• Sudden weight gain
• Numbness or tingling in your fingers, toes, or around your mouth
• Swelling of your arms, hands, legs, and ankles
• Seizures.

Notes:

Your healthcare provider may change your dose, temporarily stop, or permanently stop treatment with Cabometyx if you have certain side effects.

These are not all of the possible side effects of Cabometyx. Call your doctor for medical advice about side effects.

How does Cabometyx work?

Cabometyx (cabozantinib) works by blocking multiple tyrosine kinase enzymes, including MET, VEGFR, AXL, RET, and others. These enzymes regulate normal cell functions but also drive cancer-related processes like tumor growth, spread, blood vessel formation, drug resistance, and tumor microenvironment maintenance.

Cabometyx (cabozantinib) belongs to the drug class called multikinase inhibitors. It may also be called a VEGF/VEGFR inhibitor.

Before taking this medicine

Before you take Cabometyx, tell your healthcare provider about all of your medical conditions, including if you:

• Have had a liver problem other than liver cancer
• Have a recent history of bleeding, including coughing up or vomiting blood, or black tarry stools.
• Have an open or healing wound
• Have high blood pressure
• Have heart problems
• Have a low calcium level in your blood (hypocalcemia)
• Plan to have any surgery, dental procedure, or have had a recent surgery. You should stop taking Cabometyx at least 3 weeks before planned surgery
• Are pregnant, or plan to become pregnant
• Are breastfeeding or plan to breastfeed.

Pregnancy

Cabometyx can harm your unborn baby.

• If you can become pregnant, your healthcare provider will check your pregnancy status before you start treatment with Cabometyx.
• Females who can become pregnant should use effective birth control (contraception) during treatment and for 4 months after their last dose of Cabometyx.
• Talk to your healthcare provider about birth control methods that may be right for you.
• If you become pregnant or think you are pregnant, tell your healthcare provider right away.

Breastfeeding

It is not known if Cabometyx passes into your breast milk. Do not breastfeed during treatment and for 4 months after your last dose of Cabometyx.

How should I take Cabometyx?

• Take Cabometyx exactly as your healthcare provider tells you to take it.
• Do not take Cabometyx with food. Take Cabometyx on an empty stomach at least 1 hour before or at least 2 hours after eating.
• Swallow Cabometyx tablets whole.
• Do not crush, chew, or split Cabometyx tablets.

What happens if I miss a dose?

If you miss a dose and your next scheduled dose is in less than 12 hours, take your next dose at the normal time. Do not make up the missed dose.

What should I avoid while taking Cabometyx?

Avoid drinking grapefruit juice, eating grapefruit, or taking supplements that contain grapefruit or St. John’s wort during treatment with Cabometyx.

What other drugs will affect Cabometyx?

Tell your healthcare provider about all the medicines you take, including prescription or over-the-counter medicines, vitamins, and herbal supplements. Cabometyx and certain other medicines may affect each other, causing side effects.

Do NOT substitute Cabometyx tablets with cabozantinib capsules. 

Dosing information

• Administer on an empty stomach at least 1 hour before or at least 2 hours after eating.
• Stop treatment with Cabometyx at least 3 weeks before scheduled surgery, including dental surgery.

Adult Dose of Cabometyx for RCC

• Monotherapy: 60 mg orally once daily
• Combination therapy: 40 mg orally once daily with:
  • nivolumab 240 mg by intravenous infusion every 2 weeks

OR

• • nivolumab 480 mg by intravenous infusion every 4 weeks

OR

• • nivolumab 600 mg and hyaluronidase 10,000 units subcutaneously every 2 weeks

OR

• • nivolumab 1,200 mg and hyaluronidase 20,000 units subcutaneously every 4 weeks

Adult Dose of Cabometyx for HCC

• 60 mg orally once daily

Adult and Pediatric 12+ Dose of Cabometyx for DTC, pNET, epNET

• ≥40 kg: 60 mg orally once daily
• <40 kg: 40 mg orally once daily.

Storage

Store Cabometyx at room temperature between 68°F to 77°F (20°C to 25°C).

Keep out of the reach of children.

What are the ingredients in Cabometyx?

Active ingredient: cabozantinib

Inactive ingredients: microcrystalline cellulose, lactose anhydrous, hydroxypropyl cellulose, croscarmellose sodium, colloidal silicon dioxide, and magnesium stearate. The film coating contains hypromellose, titanium dioxide, triacetin, and iron oxide yellow.

Available as 20 mg, 40 mg, 60 mg, oral tablets.

Company

Cabometyx is manufactured for Exelixis, Inc. Alameda, CA 94502.

What is Alecensa?

Alecensa (alectinib) is an oral kinase inhibitor that may be used to treat certain types of non-small cell lung cancer (NSCLC) in adults that have been caused by an abnormal anaplastic lymphoma kinase (ALK) gene, specifically:

• to help prevent your lung cancer from coming back after your tumor has been removed by surgery (adjuvant), or
• as treatment when your lung cancer has spread to other parts of your body (metastatic).

Alecensa is used only if your cancer has a specific genetic marker (an abnormal “ALK” gene). Your doctor will test you for this gene.

Alecensa works by targeting ALK and RET — these are 2 tyrosine kinases present in 5% to 7% of people with NSCLC. By preventing the activity of these kinases, Alecensa reduces the viability of cancer cells.

Alecensa was FDA approved on December 11, 2015.

Alecensa side effects

Common Alecensa side effects may include:

• anemia;
• constipation;
• swelling in your face, eyelids, hands, or lower legs;
• tired feeling; or
• muscle pain.

Serious side effects and warnings

Alecensa can cause serious or life-threatening side effects.

Get emergency medical help if you have signs of an allergic reaction to Alecensa, such as hives; difficulty breathing; or swelling of your face, lips, tongue, or throat.

Serious side effects of Alecensa include the following:

Liver problems (hepatotoxicity). These are common with Alecensa and can be severe. Your healthcare provider will do blood tests at least every 2 weeks for the first 3 months, and then 1 time each month and
as needed during treatment to check your liver function. Tell your healthcare provider right away if you get any of the following signs and symptoms:

• feeling tired
• feeling less hungry than usual
• yellowing of your skin or the whites of your eyes
• dark urine
• itchy skin
• nausea or vomiting
• pain on the right side of your stomach area
• bleeding or bruising more easily than normal.

Lung problems, which may be severe or life-threatening and similar to those symptoms from lung cancer. Tell your healthcare provider right away if you have any new or worsening symptoms, including trouble breathing, shortness of breath, cough, or fever.

Kidney problems, which may be fatal. Tell your healthcare provider right away if you have a change in the amount or color of your urine, or if you get new or worsening swelling in your legs or feet.

Slow heartbeat (bradycardia). Alecensa may cause very slow heartbeats that can be severe. Your healthcare
provider will check your heart rate and blood pressure during treatment. Tell your healthcare
provider right away if you feel dizzy, lightheaded, or if you faint during treatment. Tell your
healthcare provider if you take any heart or blood pressure medicines.

Severe muscle pain, tenderness, and weakness (myalgia). Muscle problems are common with Alecensa and can be severe. Your healthcare provider will do blood tests at least every 2 weeks for the first month and as needed during treatment. Tell your healthcare provider right away if you get new or worsening signs and
symptoms of muscle problems, including unexplained muscle pain or muscle pain that does not go away,
tenderness, or weakness.

Breakdown of healthy red blood cells earlier than normal (hemolytic anemia). Hemolytic anemia can happen in some people who take Alecensa. If this happens, you may not have enough healthy red blood cells. Your
healthcare provider may temporarily stop treatment and do blood tests, if needed, to check for this problem. If you develop hemolytic anemia, your healthcare provider may either restart you at a lower dose when the
hemolytic anemia goes away or may stop your treatment. Tell your healthcare provider right away
if you experience yellow skin (jaundice), weakness or dizziness, or shortness of breath.

Call your doctor at once if you have:

• heart problems–severe dizziness, very slow heartbeats;
• a light-headed feeling, like you might pass out;
• unexplained muscle pain, tenderness, or weakness;
• lung problems – sudden chest pain or discomfort, wheezing, dry cough or cough with mucus, feeling short of breath;
• low red blood cells (anemia) – pale skin, unusual tiredness, feeling light-headed, cold hands and feet;
• kidney problems – a change in your urine color, little or no urination, swelling in your feet or ankles; or
• liver problems – stomach pain (upper right side), loss of appetite, easy bruising or bleeding, feeling tired, dark urine, jaundice (yellowing of the skin or eyes).

Your cancer treatments may be delayed or permanently discontinued if you have certain side effects.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects.

Before taking this medicine

You should not use Alecensa if you are allergic to alectinib or any of the inactive ingredients in Alecensa.

Tell your doctor if you have ever had:

• lung or breathing problems other than lung cancer;
• liver disease; or
• slow heartbeats.

Tell your healthcare provider if you are pregnant, intending to become pregnant, or breastfeeding.

Pregnancy

Alecensa can harm an unborn baby or cause birth defects if the mother or the father is taking this medicine.

• If you are a woman, do not use Alecensa if you are pregnant. Use effective birth control to prevent pregnancy while you are using this medicine and for at least 1 week after your last dose.
• If you are a man, use effective birth control if your sex partner is able to get pregnant. Keep using birth control for at least 3 months after your last dose.
• Tell your doctor right away if a pregnancy occurs while either the mother or the father is using Alecensa.

Breastfeeding

It is not known whether Alecensa passes into breast milk or if it could harm a nursing baby. You should not breastfeed while you are using alectinib and for at least 1 week after your last dose.

How should I take Alecensa?

Before you start treatment with Alecensa, your doctor will perform a special test to make sure Alecensa is the best treatment for your type of lung cancer. Follow all directions on your prescription label and read all medication guides or instruction sheets. Your doctor may occasionally change your dose. Use the medicine exactly as directed.

The usual dosage of Alecensa is 600 mg twice daily.

• Alecensa works best if you take it with food.
• Swallow the capsule whole and do not crush, chew, dissolve, or open it.
• If you vomit shortly after taking Alecensa, do not take another dose. Wait until your next scheduled dose time to take the medicine again.

You may need frequent medical tests to be sure this medicine is not causing harmful effects. Your cancer treatments may be delayed based on the results.

Store at room temperature away from moisture, heat, and light.

Alecensa dosing

Usual Alecensa Adult Dose for Non-Small Cell Lung Cancer:

• 600 mg orally 2 times a day
  • Available as Alecensa 150 mg capsules.
  • Four capsules (4 x 150 mg) make up one dose of Alecensa to be taken twice a day.

Duration of Therapy: Until disease progression or unacceptable toxicity

Comments: This drug should be taken with food.

What happens if I miss a dose?

Skip the missed dose and use your next dose at the regular time. Do not use two doses at one time. If you vomit shortly after taking Alecensa, do not take another dose. Wait until your next scheduled dose time to take the medicine again.

What to avoid

Alectinib could make you sunburn more easily. Avoid sunlight or tanning beds during treatment and for at least 7 days after your last dose of Alecensa. Wear protective clothing and use sunscreen (SPF 50 or higher) when you are outdoors.

What is Gavreto?

Gavreto (pralsetinib) is an oral prescription medicine that may be used to treat the following cancers caused by abnormal RET (rearranged during transfection) genes that have spread (metastasized):

• non-small cell lung cancer (NSCLC) in adults
• advanced thyroid cancer or thyroid cancer in adults and children 12 years of age and older who require a medicine by mouth or injection (systemic therapy) and who have received radioactive iodine and it did not work or is no longer working.

Your healthcare provider will perform a test to make sure that Gavreto is right for you.

Gavreto is a targeted cancer treatment that works by blocking the activity of a specific protein called RET, which is involved in the growth and development of certain cancers. Gavreto’s mechanism of action means it effectively targets RET-driven cancers, including those with specific mutations or fusions.

Gavreto gained FDA approval initially under the accelerated designation for metastatic RET fusion-positive NSCLC on September 4, 2020, which was upgraded to full approval on August 9, 2023.

• FDA approval for metastatic RET fusion-positive thyroid cancer for Gavreto is still under the accelerated approval designation. Continued approval may be dependent on favorable clinical trials.

Gavreto side effects

The most common side effects of Gavreto are:

• muscle or joint pain
• constipation or diarrhea
• high blood pressure
• low blood cell counts or other abnormal laboratory tests
• feeling tired
• swelling of your face, arms, legs, hands, and feet (edema)
• fever
• cough
• low sodium levels and other electrolyte changes.

The most common severe abnormal blood test results with Gavreto include:

• decreased white blood cell, red blood cell, and platelet counts
• decreased levels of phosphate, body salt (sodium), calcium, and potassium in the blood
• abnormal liver function blood tests
• increased levels of an enzyme called alkaline phosphatase in the blood (this is a test for liver or bone problems)
• increased levels of potassium in the blood.

Serious side effects and warnings

Gavreto may cause the following serious side effects.

Lung problems. Gavreto may cause severe or life-threatening inflammation of the lungs during treatment,
which can lead to death. Tell your healthcare provider right away if you have any new or worsening
symptoms, including:

• shortness of breath
• cough
• fever.

High blood pressure (hypertension). High blood pressure is common with Gavreto and may sometimes
be severe. You should check your blood pressure regularly during treatment. Tell your healthcare provider if you have increased blood pressure readings or get any symptoms of high blood pressure, including:

• confusion
• headaches
• shortness of breath
• dizziness
• chest pain.

Liver problems. Liver problems (showing as increased liver function blood test results) can happen during treatment with Gavreto and may sometimes be serious. Your healthcare provider will do blood tests before and
during treatment to check you for liver problems. Tell your healthcare provider right away if you get any signs or symptoms of liver problems during treatment, including:

• yellowing of your skin or the white part of your eyes (jaundice)
• dark “tea-colored” urine
• sleepiness
• bleeding or bruising
• loss of appetite
• nausea or vomiting
• pain on the upper right side of your stomach area.

Bleeding problems. Gavreto can cause bleeding which can be serious and cause death. Tell your
healthcare provider if you have any signs or symptoms of bleeding during treatment, including:

• vomiting blood or if your vomit looks like coffee grounds
• pink or brown urine
• red or black (looks like tar) stools
• coughing up blood or blood clots
• unusual bleeding or bruising of your skin
• menstrual bleeding that is heavier than normal
• unusual vaginal bleeding
• nose bleeds that happen often
• drowsiness or difficulty being awakened
• confusion
• headache
• change in speech.

Tumor lysis syndrome (TLS). TLS is caused by a fast breakdown of cancer cells. TLS can cause you to
have kidney failure and the need for dialysis treatment, an abnormal heartbeat, and may sometimes lead
to hospitalization. Your healthcare provider may do blood tests to check you for TLS. You should stay well
hydrated during treatment with Gavreto. Call your healthcare provider or get emergency medical help
right away if you develop any of these symptoms during treatment:

• nausea
• vomiting
• weakness
• swelling
• shortness of breath
• muscle cramps
• seizures.

Risk of wound healing problems. Wounds may not heal properly during treatment with Gavreto. Tell your
healthcare provider if you plan to have any surgery before or during treatment.

• You should not take Gavreto for at least 5 days before surgery.
• Your healthcare provider should tell you when you may start taking Gavreto again after surgery

Gavreto may affect fertility in males and females, which may affect your ability to have children. Talk to your
healthcare provider if this is a concern for you.

Allergic reactions. Gavreto may cause hypersensitivity reactions. Get emergency medical help if you have signs of an allergic reaction to Gavreto such as hives; difficulty breathing; or swelling of your face, lips, tongue, or throat.

Gavreto may affect the growth plates of growing children.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to the FDA .

Before taking

Before taking Gavreto, tell your healthcare provider about all of your medical conditions, including if you:

• have lung or breathing problems other than lung cancer
• have high blood pressure
• have bleeding problems
• have liver problems
• plan to have surgery. You should stop taking Gavreto at least 5 days before your planned surgery
• are pregnant or plan to become pregnant
• are breastfeeding or plan to breastfeed.

Gavreto may impair fertility. Talk to your healthcare provider if this is a concern for you.

It is not known if Gavreto is safe and effective when used to treat cancers caused by abnormal RET genes:

• in children for the treatment of NSCLC
• in children younger than 12 years of age for the treatment of thyroid cancer.

Pregnancy

Gavreto can harm an unborn baby. If you are a female who can become pregnant your healthcare provider will do a pregnancy test before you start treatment with Gavreto. You should use an effective form of non-hormonal birth control (contraception) during treatment and for 2 weeks after your last dose of Gavreto.

Birth control methods that contain hormones (such as birth control pills, injections, or transdermal system
patches) may not work as well during treatment with Gavreto. To prevent pregnancy while using Gavreto, use a barrier form of birth control: condom, diaphragm, cervical cap, or contraceptive sponge. Talk to your healthcare provider about birth control methods that may be right for you during this time.

Tell your healthcare provider right away if you become pregnant or think you might be pregnant during
treatment with Gavreto.

Males with female partners who can become pregnant should use effective birth control (contraception) during treatment with Gavreto and for 1 week after their last dose.

Breastfeeding

It is not known if Gavreto passes into your breast milk. Do not breastfeed during treatment and for 1 week after your last dose of Gavreto.

How should I take Gavreto?

Take Gavreto exactly as your healthcare provider tells you to take it. Do not change your dose or stop using the medicine without your doctor’s advice.

• Take Gavreto 1 time each day.
• Gavreto should be taken on an empty stomach at least 1 hour before or 2 hours after a meal.

Your healthcare provider may change your dose, temporarily stop, or permanently stop treatment with Gavreto if you develop side effects.

• If you vomit after taking a dose of Gavreto, do not take an extra dose. Take your next dose of Gavreto at your regular time the next day.
• Your blood pressure will need to be checked often and you will need frequent blood tests.
• Your doctor will monitor your liver function (specifically ALT and AST enzymes) before initiating Gavreto, then every 2 weeks for the first 3 weeks, then monthly thereafter.
• If you need surgery, tell your surgeon you currently use this medicine. You may need to stop for a short time.

Gavreto dosing information

Gavreto dosage for adults and children 12 and over: Gavreto 400 mg orally 1 time a day.

• Each Gavreto capsule is 100 mg, which means you will take 4 capsules of Gavreto.
• Gavreto should be taken on an empty stomach, at least 1 hour before or 2 hours after a meal.
• If you vomit shortly after taking Gavreto, do not take another dose. Wait until your next scheduled dose time to take the medicine again.

What happens if I miss a dose?

If you miss a dose of Gavreto, take it as soon as possible on the same day. Then take your next dose of
Gavreto at your regular time the next day. Skip the missed dose if it is almost time for your next dose. Do not take two doses at one time.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line .

What to avoid

Gavreto can delay wound healing. Do not take Gavreto for at least 5 days before surgery. Your healthcare provider should tell you when you may start taking Gavreto again after surgery

What other drugs will affect Gavreto?

Sometimes it is not safe to use certain medications at the same time. Some drugs can affect your blood levels of other drugs you take, which may increase side effects or make the medications less effective. Tell your doctor about all your current medicines including prescription medicines, over-the-counter drugs, vitamins, and herbal products. Many drugs can interact with Gavreto, especially:

• some antibiotic or antifungal medicines
• antiviral medications used to treat HIV or hepatitis C
• some cancer medications
• certain cholesterol medications
• seizure medications.

Avoid administering Gavreto with strong or moderate CYP3A inhibitors and/or P-gp inhibitors such as clarithromycin, erythromycin, diltiazem, itraconazole, ketoconazole, ritonavir, verapamil, goldenseal, and grapefruit. Your doctor may reduce the dosage of Gavreto if coadministration is unavoidable.

Avoid administering Gavreto with strong or moderate CYP3A inducers such as phenobarbital, phenytoin, rifampicin, St. John’s Wort, and glucocorticoids. Your doctor may increase the dosage of Gavreto if coadministration is unavoidable.

This list is not complete and many other drugs may interact with pralsetinib. This includes prescription and over-the-counter medicines, vitamins, and herbal products. Not all possible drug interactions are listed here. See the Gavreto Prescribing Information for a complete list.

Storage

Store Gavreto 100 mg capsules at room temperature between 68°F to 77°F (20°C to 25°C) away from moisture and heat.

Keep out of the reach of children.

Gavreto ingredients

Active ingredient: pralsetinib 100 mg.

Inactive ingredients: citric acid, hydroxypropyl methylcellulose (HPMC), magnesium stearate, microcrystalline cellulose (MCC), pregelatinized starch, and sodium bicarbonate.

Capsule shell: FD&C Blue #1 (Brilliant Blue FCF), hypromellose, and titanium dioxide.

White printing ink: butyl alcohol, dehydrated alcohol, isopropyl alcohol, potassium hydroxide, propylene glycol, purified water, shellac, strong ammonia solution, and titanium dioxide.

Gavreto 100 mg capsules are available in bottles of 60, 90, and 120 capsules.

What is Gomekli?

Gomekli (mirdametinib) is an FDA-approved medication used to treat neurofibromatosis type 1 (NF1) with plexiform neurofibromas causing symptoms and cannot be surgically removed. Gomekli helps to reduce tumor size, relieve symptoms, and improve quality of life. Gomekli can be used by adults and patients 2 years and older. Neurofibromatosis type 1 with plexiform neurofibromas is often shortened to NF1-PN.

Gomekli is from the drug class MEK inhibitors. 

Gomekli mechanism of action is by inhibiting proteins MEK1 and MEK2 which are part of the messenger system that controls cell growth and division.  Inhibiting MEK proteins controls cell division and helps slow cancer growth.

Gomekli FDA approval was received for treatment of neurofibromatosis type 1 (NF1) in adults and pediatric patients (2 years and older) who have symptomatic plexiform neurofibromas (PN) that are not able to be completely removed by surgery. Gomekli FDA approval was granted to SpringWorks Therapeutics on February 11, 2025, after positive results from the Phase 2b ReNeu trial (NCT03962543).

Gomekli tablets (1mg) are taken twice daily, they can be swallowed whole or maybe dispersed in drinking water and taken orally as a liquid. 

What is Neurofibromatosis Type 1?

Neurofibromatosis Type 1 (NF1) is a rare genetic disorder due to a mutation in the NF1 gene. The NF1 gene has a role in signaling cell growth and survival, and when there is a mutation this causes excessive cell growth which results in tumors. 

NF1 patients have tumors called plexiform neurofibromas (PN), that grow along the peripheral nerve sheath and can cause severe disfigurement, pain, and functional impairment. Often the plexiform neurofibromas are unable to be surgically removed due to the infiltrative tumor growth pattern along nerves. These plexiform neurofibromas have the potential to become malignant peripheral nerve sheath tumors, which can be aggressive and potentially fatal.

How well does Gomekli work?

In the clinical trial ReNeu (Phase 2b ReNeu trial) Gomekli’s efficacy was determined by its confirmed overall response rate (ORR), measured by a blinded independent central review using volumetric MRI analysis. The results from the ReNeu trial demonstrated:

• 41% overall response rate* in adults 
• 52% overall response rate* in pediatric patients 
• Tumor volume reductions were deep and durable, with a median best percentage change of -41% in adults and -42% in children
• 88% of adults and 90% of children who achieved a confirmed response maintained that response for at least 12 months
• 50% of adults and 48% of children had a response lasting 24 months or longer

Additionally, patients experienced early and sustained improvements in pain and quality of life, as assessed across multiple patient-reported outcome tools.

*Overall response rate (ORR) is the percentage of patients who achieve either a complete response (disappearance of the target tumor) or a partial response (a 20% or greater reduction in tumor size). 

Gomekli side effects

Common Gomekli side effects

Common adult Gomekli side effects included:

• Rash (90%)
• Diarrhea (59%)
• Nausea (52%)
• Muscle and joint pain (41%)
• Vomiting (38%)
• Tiredness or fatigue (29%)
• COVID-19 (22%)
• Peripheral neuropathy (21%)

The most common laboratory abnormalities in Grade 3 or 4 (>2%) were decreased neutrophil count and increased creatine phosphokinase.

Common pediatric Gomekli side effects included:

• Rash (73%)
• Diarrhea (55%)
• Muscle and joint pain (41%)
• Abdominal pain (39%) 
• Vomiting (39%)
• Headache (34%)
• Paronychia (32%)
• Left ventricular dysfunction (27%),
• Nausea (27%).
• Mouth ulcers (20%)
• Fever (20%)
• COVID 19 (10%)
• Upper respiratory tract infection – URTI (23%)

The most common Grade 3 or 4 laboratory abnormalities (>2%) were decreased neutrophil count and increased creatine phosphokinase.

These side effects occurred in patients in the Phase 2b ReNeu clinical trial.

Serious side effects and warnings

Gomekli carries significant safety warnings, including:

• Ocular Toxicity: May cause retinal vein occlusion (RVO), retinal pigment epithelium detachment (RPED), and blurred vision. Ophthalmic assessments are required before and during treatment.
• Left Ventricular Dysfunction: Gomekli can lead to decreased ejection fraction, with echocardiography recommended for starting treatment, every 3 months for the first year and then as often as directed. 
• Dermatologic Adverse Reactions: Rash and other skin-related issues were common, requiring supportive care and potential dose adjustments, or stopping of treatment.
• Embryo-Fetal Toxicity: Gomekli may cause fetal harm; pregnancy status must be verified before starting treatment. Effective contraception is recommended for patients of reproductive potential.

These are not all of the possible side effects of Gomekli. Call your doctor for medical advice about side effects. You may report side effects to the FDA .

Before taking this medicine.

Tell your healthcare provider about all of your medical conditions, including if you:

• have eye problems
• have heart problems
• are pregnant or plan to become pregnant as Gomekli can harm your unborn baby.

Pregnancy

Females who are able to become pregnant:

• You should have a pregnancy test to confirm you are not pregnant before starting treatment with this medicine.
• Use effective birth control (contraception) during treatment and for 6 weeks after your last dose.
• Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment

Males with female partners who are able to become pregnant:

• Use effective birth control (contraception) during treatment with Gomekli and for 3 months after your last dose.
• Tell your healthcare provider right away if your female partner becomes pregnant or thinks she may be pregnant during treatment or within the 3 months after stopping treatment with this medicine.

Breastfeeding

• Tell your healthcare provider if you are breastfeeding or plan to breastfeed, as it is not known if Gomekli passes into your breastmilk.
• Do not breastfeed during treatment with GOMEKLI and for 1 week after your last dose.
• Talk to your healthcare provider about the best way to feed your baby during this time.

How should I take Gomekli?

• Gomekli is taken twice a day, about 12 hours apart, for 21 days, followed by 7 days off treatment, to complete a 28-day treatment cycle. Your healthcare provider will decide how many treatment cycles are right for you.
• Gomekli can be taken with or without food.
• Gomekli is available as capsules and tablets for oral suspension.

How to take Gomekli capsules:

• Swallow each capsule whole with drinking water. If more than 1 capsule is required, swallow 1 capsule at a time
• You should not open, break, or chew the capsules

How to take Gomekli tablets

• You can swallow each tablet for oral suspension whole with drinking water. If more than 1 tablet is required, swallow 1 tablet at a time.
• You can disperse the tablets for oral suspension in drinking water to make a liquid (suspension) that you swallow. See full directions on how to prepare and take Gomekli as an oral suspension.

 Take Gomekli exactly as your healthcare provider tells you to take it. Your healthcare provider may change your dose, temporarily stop, or permanently stop treatment with Gomekli if you develop certain side effects.

Your healthcare provider will decide how many treatment cycles are right for you.

Gomekli dosing information 

Gomekli recommended dosage is 2 mg/m2 orally twice daily (approximately every 12 hours) with or without food for the first 21 days of each 28-day cycle.

• The dose may be reduced due to adverse reactions.
• The maximum dose is 4 mg twice daily. 

Gomekli SpringWorks CareConnections

SpringWorks Therapeutics has established SpringWorks CareConnections, a support program for  NF1-PN patients prescribed Gomekli, offering assistance with insurance coverage, financial support, and educational resources. Gomekli is expected to be available in the United States through a specialty pharmacy and distributor network.

What happens if I miss a dose?

If you miss a dose of your tablets or capsules, skip the missed dose and take your next dose at your regularly scheduled time.

If you vomit at any time after taking your dose, do not take an additional dose. Take your next dose at your regularly scheduled time.

What other drugs will affect this medicine?

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.This list of interactions is not complete. 

Gomekli Package Insert

Review the Gomekli Package Insert for more detailed information about this medicine. The Gomekli Package Insert contains more comprehensive information on Indications and Usage, Dosage and Administration, Clinical Pharmacology, Clinical Studies, Drug Interaction, and more. Discuss any medical questions you have with your doctor or other health care provider. This is not all the information you need to know about this medicine for safe and effective use, and it does not take the place of talking to your doctor about your treatment.

The Package Insert is sometimes called Gomekli Prescribing Information (PI) or the FDA label.

Ingredients 

Active ingredient 

• mirdametinib

Inactive ingredients 

Capsules (1 mg and 2 mg) gelatin capsule

• Croscarmellose sodium, magnesium stearate, and microcrystalline cellulose. 
• The gelatin capsule shell contains FD&C blue #1, gelatin, titanium dioxide, and yellow iron oxide. The capsule is imprinted with white ink that contains butyl alcohol, dehydrated alcohol, isopropyl alcohol, potassium hydroxide, propylene glycol, purified water, shellac, strong ammonia solution, and titanium dioxide.

Tablets 1 mg tablets for oral suspension 

• Croscarmellose sodium, magnesium stearate, microcrystalline cellulose, grape flavor, and sucralose. The grape flavor includes corn syrup solids, modified corn starch, and triacetin. 

Company 

Govmekli  SpringWorks Therapeutics, Inc. 

What is Iclusig?

Iclusig is an oral kinase inhibitor that may be used to treat adults with:

• Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) in those who cannot receive any other kinase inhibitor medicines or have a specific type of abnormal gene (T315I-positive Ph+ ALL)
• Newly diagnosed Ph+ ALL in combination with chemotherapy (this approval is under the accelerated approval designation and is based on minimal residual disease (MRD)-negative complete remission (CR) at the end of induction)
• Chronic phase chronic myeloid leukemia (CML) who did not tolerate or no longer benefit from treatment with at least 2 prior kinase inhibitor medicines
• T315I-positive CML (chronic phase, accelerated phase, or blast phase).

Iclusig is not for use to treat people with newly diagnosed chronic phase CML.

It is not known if Iclusig is safe and effective in children.

Warnings

Iclusig may cause heart or blood vessel problems that could lead to heart attack, stroke, or death.

Call your doctor or get emergency medical help if you have: chest pain spreading to your jaw or shoulder, shortness of breath, dizziness, severe stomach pain, swelling in your legs, sudden numbness or weakness, headache, or problems with vision or speech.

Iclusig may also harm your liver. Call your doctor right away if you have upper stomach pain with loss of appetite, dark urine, bruising, or yellowing of your skin or eyes.

Before taking this medicine

You should not use Iclusig if you are allergic to ponatinib.

To make sure Iclusig is safe for you, tell your doctor if you have ever had:

• heart failure, high blood pressure, heart rhythm problems;
• long QT syndrome (in you or a family member);
• a stroke or blood clot;
• diabetes;
• high cholesterol;
• liver disease;
• bleeding problems;
• lactose intolerance (tablets may contain lactose);
• pancreatitis; or
• if you’ve had surgery within the past 2 weeks.

You may need to have a negative pregnancy test before starting this treatment.

Ponatinib may harm an unborn baby. Use effective birth control to prevent pregnancy while you are using this medicine and for at least 3 weeks after your last dose.

This medicine may affect fertility (ability to have children) in women. However, it is important to use birth control to prevent pregnancy because Iclusig can harm an unborn baby.

You should not breastfeed while using Iclusig and for at least 6 days after your last dose.

How should I take Iclusig?

Take Iclusig exactly as prescribed by your doctor. Follow all directions on your prescription label and read all medication guides or instruction sheets. Your doctor may occasionally change your dose.

Iclusig is usually taken once daily, with or without food.

Swallow the tablet whole and do not crush, chew, or break it.

You may need frequent medical tests to be sure this medicine is not causing harmful effects. Your cancer treatments may be delayed based on the results.

If you need surgery, tell the surgeon ahead of time that you are using Iclusig. You may need to stop using the medicine at least 1 week before surgery.

Do not stop taking Iclusig or change your medication dose without your doctor’s advice.

Store at room temperature away from moisture and heat.

Dosing information

Usual Adult Dose for Chronic Myelogenous Leukemia:

Chronic phase chronic myeloid leukemia (CP-CML):
-Initial Dose: 45 mg orally once a day
-Upon achievement of 1% BCR-ABL1 or less (standardized according to International Scale): 15 mg orally once a day
-For loss of response: Re-escalate to a previously tolerated dosage of 30 mg or 45 mg orally once a day

Accelerated phase chronic myeloid leukemia (AP-CML), blast phase chronic myeloid leukemia (BP-CML) or Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL):
-Initial dose: 45 mg orally once a day

Comments:
-The optimal dose has not been identified for AP-CML, BP-CML, and Ph+ ALL.
-Consider dose reduction for AP-CML patients who have achieved major cytogenic response.
-Continue treatment until loss of response or unacceptable toxicity.
-Drug discontinuation should be considered if patient response has not occurred by 3 months (90 days).
-This drug is not indicated and is not recommended for the treatment of patients with newly diagnosed chronic phase chronic myeloid leukemia (CP-CML).

Uses:
-For the treatment of adult patients with CP-CML with resistance or intolerance to at least two prior kinase inhibitors
-For the treatment of adult patients with AP-CML, or BP-CML or Ph+ ALL for whom no other kinase inhibitors are indicated
-For the treatment of adult patients with T315I-positive CML (chronic phase, accelerated phase, or blast phase) or T315I-positive Ph+ ALL

Usual Adult Dose for Acute Lymphoblastic Leukemia:

Chronic phase chronic myeloid leukemia (CP-CML):
-Initial Dose: 45 mg orally once a day
-Upon achievement of 1% BCR-ABL1 or less (standardized according to International Scale): 15 mg orally once a day
-For loss of response: Re-escalate to a previously tolerated dosage of 30 mg or 45 mg orally once a day

Accelerated phase chronic myeloid leukemia (AP-CML), blast phase chronic myeloid leukemia (BP-CML) or Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL):
-Initial dose: 45 mg orally once a day

Comments:
-The optimal dose has not been identified for AP-CML, BP-CML, and Ph+ ALL.
-Consider dose reduction for AP-CML patients who have achieved major cytogenic response.
-Continue treatment until loss of response or unacceptable toxicity.
-Drug discontinuation should be considered if patient response has not occurred by 3 months (90 days).
-This drug is not indicated and is not recommended for the treatment of patients with newly diagnosed chronic phase chronic myeloid leukemia (CP-CML).

Uses:
-For the treatment of adult patients with CP-CML with resistance or intolerance to at least two prior kinase inhibitors
-For the treatment of adult patients with AP-CML, or BP-CML or Ph+ ALL for whom no other kinase inhibitors are indicated
-For the treatment of adult patients with T315I-positive CML (chronic phase, accelerated phase, or blast phase) or T315I-positive Ph+ ALL

What happens if I miss a dose?

Take the medicine as soon as you can, but skip the missed dose if it is almost time for your next dose. Do not take two doses at one time.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line .

What to avoid

Grapefruit may interact with ponatinib and lead to unwanted side effects. Avoid the use of grapefruit products.

Iclusig side effects

Get emergency medical help if you have signs of an allergic reaction to Iclusig: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Iclusig may cause heart or blood vessel problems that could lead to heart attack or stroke. Call your doctor right away or get emergency medical help if you have:

• heart attack symptoms – chest pain or pressure, pain spreading to your jaw or shoulder, feeling short of breath;
• signs of a stroke – sudden numbness or weakness (especially on one side of the body), sudden severe headache, slurred speech, problems with vision or balance; or
• signs of a blood clot – severe stomach pain, pain or swelling in your arms or legs, coughing up blood.

Call your doctor at once if you have:

• dizziness, confusion, headache, change in mental status;
• a seizure;
• swelling, rapid weight gain, feeling short of breath;
• a wound that will not heal;
• eye problems – vision problems, eye pain or swelling, bleeding in the eye, increased sensitivity to light, flashes of light or “floaters” in your vision;
• heart problems – chest pain, shortness of breath, fast or pounding heartbeats, feeling like you might pass out;
• a hole or tear in your stomach or intestine – severe pain or swelling in your stomach with a high fever;
• low blood cell counts – fever, chills, tiredness, mouth sores, skin sores, easy bruising, unusual bleeding, pale skin, cold hands and feet;
• nerve problems – muscle weakness, trouble moving your eyes or other parts of your face, tingling, burning pain, numbness in your hands and feet;
• severe bleeding – nosebleeds, bloody or tarry stools, pink or brown urine, heavy menstrual periods, coughing up blood or vomit that is bloody or looks like coffee grounds; or
• signs of liver or pancreas problems – loss of appetite, upper stomach pain (that may spread to your back), nausea or vomiting, fast heart rate, dark urine, jaundice (yellowing of the skin or eyes).

Common Iclusig side effects include:

• dry skin, rash;
• stomach pain, nausea, vomiting, diarrhea, constipation;
• headache, muscle or joint pain;
• pain in your arms, hands, legs, or feet;
• increased blood pressure; or
• fever, tired feeling.

What is Inlyta?

Inlyta is a prescription medicine that interferes with the growth of some cancer cells.

Inlyta is used to treat advanced kidney cancer (advanced renal cell carcinoma).

Inlyta is sometimes given after at least one other cancer treatment did not work or has stopped working.

It is not known if Inlyta is safe and effective in children.

Warnings

Do not use Inlyta if you are pregnant. It could harm the unborn baby. Use birth control to prevent pregnancy while you are receiving this medicine, whether you are a man or a woman. Inlyta use by either parent may cause birth defects or miscarriage.

Axitinib can increase your risk of serious bleeding. Stop using Inlyta and call your doctor at once if you have severe stomach pain, bloody or tarry stools, coughing up blood, or any heavy or unusual bleeding.

Some people taking this medicine have developed a perforation (a hole or tear) or a fistula (an abnormal passageway) within the stomach or intestines. Call your doctor if you have severe stomach pain, or if you feel like you are choking and gagging when you eat or drink.

Inlyta is usually taken once every 12 hours. It should be taken at evenly spaced intervals. Follow your doctor’s instructions.

To be sure this medication is not causing harmful effects, your kidney, thyroid, and liver function will need to be tested. Your blood pressure will also need to be checked often. Visit your doctor regularly.

Before taking this medicine

You should not use Inlyta if you are allergic to axitinib.

To make sure Inlyta is safe for you, tell your doctor if you have:

• a perforation (a hole or tear) in your stomach or intestines;
• bleeding problems, or a wound that has not healed;
• a heart attack, heart failure, stroke, or blood clot;
• high blood pressure;
• liver disease; or
• a thyroid disorder.

You may need to have a negative pregnancy test before starting this treatment.

Both men and women using this medicine should use effective birth control to prevent pregnancy. Axitinib can cause harm an unborn baby if the mother or father is using this medicine.

Keep using birth control for at least 1 week after your last dose. Tell your doctor right away if a pregnancy occurs while either the mother or the father is using Inlyta.

This medicine may affect fertility (ability to have children) in both men and women. However, it is important to use birth control to prevent pregnancy because axitinib can harm an unborn baby.

It is not safe to breast-feed a baby while you are using this medicine. Also do not breast-feed for at least 2 weeks after your last dose.

How should I take Inlyta?

Take Inlyta exactly as prescribed by your doctor. Follow all directions on your prescription label and read all medication guides or instruction sheets. Your doctor may occasionally change your dose.

Inlyta should be taken at evenly spaced intervals, usually once every 12 hours.

Take this medicine with a full glass of water. You may take this medicine with or without food.

Swallow the tablet whole and do not crush, chew, or break it.

If you vomit shortly after taking Inlyta, do not take another dose. Wait until your next scheduled dose time to take the medicine again.

Your blood pressure will need to be checked often. You may also need frequent medical tests.

If you need surgery, tell your surgeon you currently use Inlyta. You will need to stop using the medicine at least 24 hours before a planned surgery.

Store at room temperature away from moisture and heat.

Dosing information

Usual Adult Dose for Renal Cell Carcinoma:

Initial dose: 5 mg orally twice a day
Maintenance dose: Increase or decrease dose based on individual safety and tolerability
Dose range: 2 to 10 mg twice a day

Comments: Doses should be taken approximately 12 hours apart.

Use: For the treatment of advanced renal cell carcinoma after the failure of 1 prior systemic therapy.

What happens if I miss a dose?

Skip the missed dose and use your next dose at the regular time. Do not use two doses at one time.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line .

What to avoid

Grapefruit may interact with axitinib and lead to unwanted side effects. Avoid the use of grapefruit products.

Inlyta side effects

Get emergency medical help if you have signs of an allergic reaction to Inlyta: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Some people taking Inlyta have developed a perforation (a hole or tear) or a fistula (an abnormal passageway) within the stomach or intestines. Call your doctor if you have severe stomach pain, or if you feel like you are choking and gagging when you eat or drink.

Also call your doctor at once if you have:

• chest pain or pressure, pain spreading to your jaw or shoulder, trouble breathing;
• sudden numbness or weakness, problems with vision or speech;
• headache, confusion, thinking problems, seizure (convulsions);
• a light-headed feeling, like you might pass out;
• easy bruising, unusual bleeding, purple or red spots under your skin;
• heavy menstrual bleeding;
• any bleeding that will not stop;
• liver problems – severe nausea or vomiting, right-sided upper stomach pain, loss of appetite, dark urine, clay- colored stools, jaundice (yellowing of the skin or eyes);
• heart problems – swelling, rapid weight gain, feeling short of breath;
• signs of stomach bleeding – bloody or tarry stools, coughing up blood or vomit that looks like coffee grounds; or
• signs of a thyroid problem – sudden weight gain or loss, feeling very weak or tired, muscle pain, feeling hot or cold, hair loss, hoarse or deepened voice.

Common Inlyta side effects may include:

• liver problems;
• stomach pain;
• muscle and bone pain;
• abnormal blood tests;
• mouth sores or swelling of the lining of the mouth, nose, eyes, throat, intestines, or vagina;
• cough, shortness of breath;
• headache;
• nausea, vomiting, diarrhea, constipation;
• rash, itching, redness, and peeling skin on your hands or feet;
• feeling weak or tired;
• increased blood pressure;
• decreased appetite, weight loss; or
• hoarse voice.

What is Inrebic?

Inrebic is used to reduce symptoms of myelofibrosis (MF) in adults, such as night sweats, itching, pain, and feeling full when eating, and it also helps reduce spleen volume. Myelofibrosis is a rare type of bone marrow disorder.

Inrebic’s mechanism of action involves blocking certain types of enzymes called kinases that are overactive in myelofibrosis, such as Janus-associated kinase 2 (JAK2) and FMS-like tyrosine kinase 3 (FLT3). These enzymes are involved in signaling pathways that contribute to excessive inflammation in bone marrow, an enlarged spleen, and symptoms of myelofibrosis. Inrebic (fedratinib) belongs to the drug class called kinase inhibitors.

Inrebic gained FDA approval on August 16, 2019. There is no Inrebic generic.

Inrebic uses

Inrebic is used to treat intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis in adults.

Inrebic side effects

The most common side effects of Inrebic are:

• diarrhea
• nausea
• low red blood cell count (anemia)
• vomiting.

Serious side effects and warnings

Inrebic carries a Boxed Warning for encephalopathy, including Wernicke’s encephalopathy.

Encephalopathy (including Wernicke’s encephalopathy). A serious and sometimes fatal neurological problem called encephalopathy (including Wernicke’s encephalopathy) has happened in some people who take Inrebic. Wernicke’s encephalopathy is a neurologic emergency that can happen if you do not have enough Vitamin B1 (thiamine) in your body. Your healthcare provider will do a blood test to check your Vitamin B1 level and treat you with Vitamin B1 if your level is too low before starting treatment with Inrebic. Your healthcare provider may also check your Vitamin B1 level during treatment and you should take a Vitamin B1 supplement (100 mg of thiamine) throughout treatment with Inrebic. Get emergency medical help right away if you develop the following:

• confusion, memory problems, or drowsiness
• problems with balance and movement, such as difficulty walking
• eye problems, such as double or blurred vision or abnormal eye movements

Call your healthcare provider right away if you experience diarrhea, nausea, vomiting, and weight loss that does not get better with treatment.

Low blood cell counts. Inrebic may cause new or worsening low red blood cell counts (anemia) and low platelet counts (thrombocytopenia) in some people. You may need a blood transfusion if your blood counts drop too low. Your healthcare provider will do blood tests to check your blood counts before you start and during treatment with Inrebic. Tell your healthcare provider if you develop any bleeding or bruising during treatment with Inrebic.

Diarrhea, nausea, and vomiting. Your healthcare provider may give you certain medicines to help treat diarrhea, nausea, and vomiting caused by Inrebic. Call your healthcare provider or get emergency medical help right away if you have diarrhea, nausea, or vomiting that does not get better with treatment.

Liver problems. Inrebic may cause increased liver enzymes. Your healthcare provider will do blood tests to check your liver function before starting and during treatment with Inrebic.

Amylase and lipase increases. You may have increases in your blood amylase or lipase levels that may indicate a problem with your pancreas. Your healthcare provider will do blood tests to check your amylase or lipase levels before starting and during treatment with Inrebic.

Major cardiac events such as heart attack, stroke, or death. An increased risk of major cardiac events has happened in people, especially those who have cardiovascular risk factors and who are current or past smokers, while taking another Janus Kinase (JAK)-inhibitor to treat rheumatoid arthritis. Get emergency help right away if you have any symptoms of a heart attack or stroke while taking Inrebic, including:

• discomfort in the center of your chest that lasts for more than a few minutes, or that goes away and comes back
• severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw
• pain or discomfort in your arms, back, neck, jaw, or stomach
• shortness of breath with or without chest discomfort
• breaking out in a cold sweat
• nausea or vomiting
• feeling lightheaded
• weakness in one part or on one side of your body
• slurred speech.

Blood clots. Increased risk of blood clots in the veins of the legs (deep vein thrombosis, DVT) or lungs (pulmonary embolism, PE) have happened in people taking another JAK inhibitor for rheumatoid arthritis and may be life-threatening. Tell your healthcare provider right away if you have any signs and symptoms of blood clots during treatment with Inrebic, including:

• swelling, pain, or tenderness in one or both legs
• sudden unexplained chest or upper back pain
• shortness of breath or difficulty breathing.

New (secondary) cancers. People who take another JAK-inhibitor for rheumatoid arthritis have an increased risk of new (secondary) cancers, including lymphoma and other cancers, except nonmelanoma skin cancer. People who smoke or who smoked in the past have an increased risk of new cancers.

Inrebic may cause an allergic reaction. Get emergency medical help if you have signs of an allergic reaction to Inrebic: hives, difficulty breathing, swelling of your face, lips, tongue, or throat.

Driving and using machines: While using this medicine, you may feel weak, tired, dizzy, or confused. You may have blurred vision. Before driving a vehicle or using machinery, wait to see how you feel after taking Inrebic.

Call your doctor at once if you have:

• severe or ongoing nausea, vomiting, or diarrhea (even while you are taking medication to prevent these effects);
• easy bruising, unusual bleeding, purple or red spots under your skin;
• low red blood cells (anemia) – pale skin, unusual tiredness, feeling light-headed or short of breath, cold hands and feet; or
• low white blood cell counts – fever, mouth sores, skin sores, sore throat, cough, trouble breathing.

You may need a blood transfusion if your blood counts get too low.

This is not a complete list of side effects, and others may occur. Call your doctor for medical advice about side effects. You may report side effects to the FDA .

Before taking this medicine

To make sure Inrebic is safe for you, tell your doctor if you:

• have low vitamin B1 (thiamine) levels
• have low red blood cell or platelet counts
• have or have had liver problems
• have or have had kidney problems
• are a current or past smoker
• have had a blood clot, heart attack, other heart problems, or stroke
• have or have had any other cancer
• are pregnant or plan to become pregnant
• are breastfeeding or plan to breastfeed.

Inrebic is not for use in patients under the age of 18 years.

Pregnancy

It is not known if Inrebic may harm your unborn baby.

Breastfeeding

It is not known if Inrebic passes into your breast milk. You should not breastfeed during treatment with Inrebic and for at least 1 month after your last dose. Talk to your healthcare provider about the best way to feed your baby during treatment.

How should I take Inrebic?

Take Inrebic exactly as prescribed by your doctor. Follow all directions on your Inrebic Package Insert and read all medication guides or instruction sheets. Your doctor may occasionally change your dose.

• Inrebic is taken 1 time a day.
• You may take Inrebic capsules with or without food. Taking Inrebic with a high-fat meal may help to reduce nausea and vomiting symptoms.
• Take Vitamin B1 (thiamine 100 mg) by mouth daily to prevent low Vitamin B1 levels during treatment with Inrebic.

You may be given other medications to help prevent nausea, vomiting, and diarrhea. Keep using these medicines for as long as your doctor has prescribed.

Call your doctor if you are sick with vomiting or diarrhea even while taking medications to prevent these side effects. You can easily become malnourished from vomiting and diarrhea. This can lead to low vitamin B1 levels.

Inrebic can lower your blood cell counts and affect liver function. Your blood will need to be tested often. Your treatments may be delayed based on the results.

Do not change your dose or stop taking Inrebic without your doctor’s advice.

Inrebic dosing

Usual Adult Dose of Inrebic for Myelofibrosis: Inrebic 400 mg orally once a day for patients with a baseline platelet count of 50 x 10⁹/L or greater

Management of thiamine levels and Wernicke’s encephalopathy (WE):

• Assess thiamine levels and nutritional status before starting therapy, periodically during therapy, and as indicated.
• Do not initiate this drug in patients with thiamine deficiency; replete thiamine before therapy initiation and during therapy if thiamine levels are low.
• If Wernicke’s encephalopathy is suspected, immediately discontinue therapy and initiate parenteral thiamine treatment.
• Monitor until symptoms resolve or improve and thiamine levels normalize.

What happens if I miss a dose?

Skip the missed dose and use your next dose at the regular time. Do not use two doses at one time.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line .

What to avoid

Follow your doctor’s instructions about any restrictions on food, beverages, or activity.

Inrebic may make you feel drowsy or dizzy and make it difficult for you to operate machinery or to drive. Do not drive or operate machinery or perform other hazardous tasks if Inrebic affects you in this way.

What is Jakafi?

Jakafi is used to treat certain types of myelofibrosis in adults.

Jakafi is also used to treat polycythemia vera in adults who have already taken a medicine called hydroxyurea and it did not work well enough or they could not tolerate it.

Jakafi is used to treat acute graft versus host disease (aGVHD) in adults and children 12 years of age and older who have taken corticosteroids and they did not work well enough.

Jakafi works by blocking certain enzymes in the body that affect blood cell production.

Warnings

You should not use Jakafi if you are allergic to ruxolitinib, or if you have severe kidney disease.

Before you take this medicine, tell your doctor if you have liver or kidney disease, if you are on dialysis, or if you are pregnant.

You should not breast-feed while you are using this medicine.

To be sure Jakafi is helping your condition and not causing harmful effects, your blood will need to be tested often. This will help your doctor determine the best dose for you to use. When you first start taking this medicine, your blood will need to be tested every 2 to 4 weeks. Do not miss any follow-up visits to your doctor.

Grapefruit and grapefruit juice may interact with Jakafi and can affect the amount of the medication in your blood. Discuss the use of grapefruit products with your doctor.

Follow all directions on your medicine label and package. Tell each of your healthcare providers about all your medical conditions, allergies, and all medicines you use.

Before taking this medicine

You should not use Jakafi if you are allergic to ruxolitinib.

Tell your doctor if you have ever had tuberculosis or if anyone in your household has tuberculosis. Also, tell your doctor if you’ve had or been exposed to tuberculosis, or if you recently traveled. Some infections are more common in certain parts of the world, and you may have been exposed during travel.

To make sure Jakafi is safe for you, tell your doctor if you have ever had:

• any type of infection;
• shingles (herpes zoster);
• a blood clot, stroke, heart attack, or other heart problems;
• low white or red blood cell counts;
• any type of cancer;
• are a current or past smoker;
• kidney disease (or if you are on dialysis);
• liver disease (especially hepatitis B); or
• high cholesterol or triglycerides (types of fat in the blood).

It is not known if ruxolitinib will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant.

Do not breastfeed while using Jakafi, and for at least 2 weeks after your last dose.

Jakafi is not approved for the treatment of myelofibrosis or polycythemia vera by anyone younger than 18 years old.

How should I take Jakafi?

Take Jakafi exactly as prescribed by your doctor. Follow all directions on your prescription label and read all medication guides or instruction sheets.

Your doctor may occasionally change your dose. You will need frequent medical tests to help your doctor determine the best dose.

You may take Jakafi with or without food. The Jakafi tablet can also be given through a nasogastric (NG) feeding tube.

Read and carefully follow any Instructions for Use provided with the medicine. Ask your doctor or pharmacist if you do not understand these instructions.

If you are on dialysis, Jakafi should be taken after your dialysis.

Using Jakafi may increase your risk of developing other cancers. Ask your doctor about this risk.

Store at room temperature away from moisture and heat.

You should not stop using Jakafi suddenly. Follow your doctor’s instructions about tapering your dose.

You may be given other medications to help prevent infection. Keep taking these medicines for as long as your doctor has prescribed.

Dosing information

Usual Adult Dose of Jakafi for Myeloproliferative Disorder:

Doses should be titrated based on safety and efficacy; CBC (complete blood count) and platelet counts should be performed every 2 to 4 weeks until doses are stabilized and then as clinically indicated

Initial Dose Based on Platelet Count:
-Platelets greater than 200 x 10⁹/L: 20 mg orally twice a day
-Platelets 100 x 10(9)/L to 200 x 10⁹/L: 15 mg orally twice a day
-Platelets 50 x 10⁹/L to less than 100 x 10⁹/L: 5 mg orally twice a day

Comments:
-Based on limited clinical data, long-term maintenance at 5 mg twice a day has not shown benefit; this dose should be limited to patients for whom the benefits outweigh the potential risks.
-Discontinue therapy if there is no spleen size reduction or symptom improvement after 6 months of therapy.

Uses:
-For the treatment of intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF, and post-essential thrombocythemia MF.

Usual Adult Dose of Jakafi for Graft Versus Host Disease:

Monitor complete blood counts (CBC), including platelet count and ANC, and bilirubin prior to
initiating therapy, every 2 to 4 weeks until doses are stabilized, and then as indicated clinically:

Acute Graft-Versus-Host Disease (GVHD): Initial dose: 5 mg orally 2 times a day
-Dose titration: Consider increasing dose to 10 mg orally 2 times a day after at least 3 days if the ANC (absolute neutrophil count) and platelet counts are not decreased by 50% or more relative to the first day of dosing
Duration of therapy: Consider tapering after 6 months for those with response who have stopped therapeutic doses of corticosteroids; taper by 1 dose level every 8 weeks (see comments); if signs or symptoms of GVHD recur during or after taper, consider retreatment

Chronic GVHD: Initial dose: 10 mg orally 2 times a day
-Duration of therapy: Consider tapering after 6 months for those with response who have stopped therapeutic doses of corticosteroids; taper by 1 dose level every 8 weeks (see comments); if signs or symptoms of GVHD recur during or after taper, consider retreatment

Comments:
-Dose level decreases: 10 mg twice a day to 5 mg twice a day to 5 mg once a day.
-See Dose Adjustment section for dose modification guidance for adverse reactions.

Uses: For the treatment of steroid-refractory acute GVHD and treatment of chronic GVHD after failure of 1 or 2 lines of systemic therapy.

Usual Adult Dose of Jakafi for Polycythemia Vera:

Doses should be titrated based on safety and efficacy; CBC and platelet counts should be performed every 2 to 4 weeks until doses are stabilized and then as clinically indicated

Initial dose: 10 mg orally twice a day
-Dose may be titrated based on safety and efficacy

Use: For treatment of polycythemia vera (PV) in adults who have had an inadequate response to or are intolerant of hydroxyurea.

Usual Pediatric Dose of Jakafi for Graft Versus Host Disease:

12 years or older:

Monitor complete blood counts (CBC), including platelet count and ANC, and bilirubin prior to
initiating therapy, every 2 to 4 weeks until doses are stabilized, and then as indicated clinically:

Acute Graft-Versus-Host Disease (GVHD): Initial dose: 5 mg orally 2 times a day
-Dose titration: Consider increasing dose to 10 mg orally 2 times a day after at least 3 days if the ANC (absolute neutrophil count) and platelet counts are not decreased by 50% or more relative to the first day of dosing
Duration of therapy: Consider tapering after 6 months for those with response who have stopped therapeutic doses of corticosteroids; taper by 1 dose level every 8 weeks (see comments); if signs or symptoms of GVHD recur during or after taper, consider retreatment

Chronic GVHD: Initial dose: 10 mg orally 2 times a day
-Duration of therapy: Consider tapering after 6 months for those with response who have stopped therapeutic doses of corticosteroids; taper by 1 dose level every 8 weeks (see comments); if signs or symptoms of GVHD recur during or after taper, consider retreatment

Comments:
-Dose level decreases: 10 mg twice a day to 5 mg twice a day to 5 mg once a day.
-See Dose Adjustment section for dose modification guidance for adverse reactions.

Uses: For the treatment of steroid-refractory acute GVHD and treatment of chronic GVHD after failure of 1 or 2 lines of systemic therapy in pediatric patients 12 years or older.

What happens if I miss a dose?

Take the medicine as soon as you can, but skip the missed dose if it is almost time for your next dose. Do not take two doses at one time.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line .

What should I avoid while taking Jakafi?

Grapefruit may interact with ruxolitinib and cause side effects. Avoid consuming grapefruit products.

Jakafi side effects

Get emergency medical help if you have signs of an allergic reaction to Jakafi:: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Some side effects may be similar to the symptoms of myelofibrosis. Call your doctor at once if you have:

• changes in the size, shape, or color of a mole or skin lesion;
• problems with speech, thought, vision, or muscle movement (these symptoms may start gradually and get worse quickly);
• nausea, vomiting, weakness, general ill feeling;
• cold sores around your mouth, skin sores or blisters, itching, tingling, skin rash, burning pain in your thigh or lower back;
• pain in your arms, back, neck, jaw, or stomach;
• signs of a stroke – sudden numbness or weakness, severe headache, slurred speech, problems with vision or balance;
• signs of a blood clot in the lung – chest pain, sudden cough or shortness of breath, dizziness, coughing up blood;
• signs of a blood clot deep in the body – pain, swelling, or warmth in one leg;
• heart attack symptoms – chest pain or pressure, pain spreading to your jaw or shoulder, nausea, sweating;
• signs of infection – fever, chills, sore throat, body aches, unusual tiredness, loss of appetite, bruising or bleeding;
• low blood cell counts – fever, chills, tiredness, mouth sores, skin sores, easy bruising, unusual bleeding, pale skin, cold hands and feet, feeling light-headed or short of breath; or
• signs of tuberculosis: fever, cough, night sweats, loss of appetite, weight loss, and feeling very tired.

Common Jakafi side effects may include:

• low blood cell counts;
• swelling;
• infection;
• bruising;
• diarrhea;
• dizziness; or
• headache.

What is Koselugo?

Koselugo is used to treat neurofibromatosis type 1 (NF1) in children aged 2 and older with plexiform neurofibromas that cannot be completely removed by surgery. It is an oral capsule that is taken 2 times a day.

Koselugo (selumetinib) gained FDA approval on April 10, 2020. There is no generic.

How does Koselugo work?

Koselugo’s mechanism of action involves blocking the actions of 2 enzymes, MEK1 and 2. These proteins are critical components of signalling pathways involved in cell growth and division. Clinical trials have shown this medication reduces the number, volume, and spread of neurofibroma tumors.

Koselugo (selumetinib)  belongs to the drug class called multikinase inhibitors.

Side effects

The most common side effects of Koselugo are:

• nausea, vomiting, stomach pain;
• itching;
• dry skin, acne, rash;
• redness around your fingernails;
• feeling weak or tired;
• mouth pain or soreness, swollen gums;
• muscle or bone pain;
• headache; or
• fever.

Diarrhea is a common side effect of this medicine. Tell your doctor right away the first time you have diarrhea while taking Koselugo.

Serious side effects and warnings

Koselugo may cause the following serious side effects:

• Heart problems. Clinical trials have shown that Koselugo can lower the amount of blood pumped by your heart, which is common and can also be severe. Your healthcare provider will do tests before and during treatment with Koselugo to check how well your heart is working. Tell your healthcare provider right away if you get any of the following signs or symptoms:
  • persistent coughing or wheezing
  • shortness of breath
  • swelling of your ankles and feet
  • tiredness
  • increased heart rate.
• Eye problems. Koselugo can cause eye problems that can lead to blindness. Your healthcare provider will check your vision before and during treatment with Koselugo. Tell your healthcare provider right away if you get any of the following signs or symptoms:
  • blurred vision
  • loss of vision
  • dark spots in your vision (floaters)
  • other changes to your vision.
• Severe diarrhea. Diarrhea is common with Koselugo and can also be severe. Tell your healthcare provider right away the first time that you get diarrhea during treatment with Koselugo. Your healthcare provider may give you medicine to help control your diarrhea and may tell you to drink more fluids.
• Skin rash. Skin rashes are common with Koselugo and can also be severe. Tell your healthcare provider if you get any of the following signs or symptoms:
  • a rash that covers a large area of your body
  • peeling skin
  • blisters.
• Muscle problems (rhabdomyolysis). Muscle problems are common with Koselugo and can also be severe. Treatment with Koselugo may increase levels of an enzyme in your blood called creatine phosphokinase (CPK) which may be a sign of muscle damage. Your healthcare provider should do a blood test to check your blood levels of CPK before you start taking Koselugo and during treatment. Tell your healthcare provider right away if you get any of the following signs or symptoms:
  • muscle aches or pain
  • muscle spasms and weakness
  • dark, reddish urine.

Drug interactions. Tell your healthcare provider if you take aspirin, blood thinners, or other medicines to treat blood clots. Koselugo contains vitamin E, which may increase your risk of bleeding.

Your healthcare provider may change your dose, temporarily stop, or permanently ask you to stop taking Koselugo if you have any of these side effects.

These are not all of the possible side effects of Koselugo. Call your doctor for medical advice about side effects. You may report side effects to the FDA .

Before taking

Before taking Koselugo, tell your healthcare provider about all of your medical conditions, including if you:

• have heart problems
• have eye problems
• have liver problems
• are pregnant or plan to become pregnant
• are breastfeeding or plan to breastfeed.

Koselugo should not be given to a child younger than 2 years old.

Pregnancy

Koselugo can harm an unborn baby. Your healthcare provider should check to see if you are pregnant before you begin treatment with Koselugo. Females who can become pregnant should use effective birth control (contraception) during treatment with Koselugo and for 1 week after their last dose.

Males with female partners who can become pregnant should use effective birth control (contraception) during treatment with Koselugo and for 1 week after their last dose.

Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with this medication.

Breastfeeding

It is not known if Koselugo passes into your breast milk. Do not breastfeed during treatment with Koselugo and for 1 week after your last dose. Talk to your healthcare provider about how to feed your baby during this time.

How should I take Koselugo?

Take Koselugo exactly as prescribed by your doctor. Follow all directions on your prescription label and read all medication guides or instruction sheets.

• Koselugo is a capsule that is taken 2 times a day.
• Can be taken with or without food.
• Take each dose at the same time each day, about 12 hours apart.
• Take with a full glass of water.
• Swallow the capsule whole and do not crush, chew, break, or open it. Tell your doctor if your child has trouble swallowing the capsule whole.

After taking

If you vomit shortly after taking Koselugo, do not take another dose. Wait until your next scheduled dose time to take the medicine again.

Tell your doctor if you start having loose stools (diarrhea). You may be given anti-diarrhea medicine. Keep using this medicine for as long as your doctor has prescribed.

• Drink plenty of liquids if you have diarrhea.

Koselugo can cause your heart to pump less blood than normal. Your heart function will need to be checked every 3 to 6 months while you are taking this medicine.

Koselugo can also cause eye problems that may lead to blindness. You will need frequent vision exams, and your next dose may be delayed based on the results.

Dosing information

Your healthcare provider decides the correct dose for you based on body surface area (height and weight). Your dose needs may change if you gain or lose weight, or if you are still growing.

• Do not change your dose or dosing schedule without your doctor’s advice.

Usual dose for children 2 years and older for NF1:

• Koselugo 25 mg/m² orally 2 times a day (approximately every 12 hours) until disease progression or unacceptable toxicity.

Reduce the recommended dosage to 20 mg/m² orally 2 times a day for patients with moderate hepatic impairment (Child-Pugh B).

• The dosage for use in patients with severe hepatic impairment (Child-Pugh C) has not been established. 

What is lorlatinib?

Lorlatinib is a kinase inhibitor for oral administration used to treat cancer. A kinase inhibitor is an enzyme inhibitor that can block the action of protein kinases.

Lorlatinib is used to treat a certain type of non-small cell lung cancer that has spread to other parts of the body. It is used only if your cancer has a specific genetic marker (an abnormal “ALK” gene). Your doctor will test you for this gene.

Lorlatinib is given after other cancer treatments did not work or have stopped working.

Lorlatinib was approved by the US Food and Drug Administration (FDA) on an “accelerated” basis. In clinical studies, some people responded to lorlatinib, but further studies are needed.

Warnings

Tell your doctor about all your current medicines and any you start or stop using. Many drugs can interact, and some drugs should not be used together.

Before taking this medicine

Some medicines can cause unwanted or dangerous effects when used with lorlatinib. Tell your doctor about all your current medicines and any you start or stop using.

To make sure lorlatinib is safe for you, tell your doctor if you have:

• heart problems, such as “AV block” (unless you have a pacemaker);
• lung disease, breathing problems;
• liver disease;
• depression;
• seizures; or
• high cholesterol or triglycerides (a type of fat in the blood).

Lorlatinib can harm an unborn baby or cause birth defects if the mother or the father is using this medicine.

• If you are a woman, do not use lorlatinib if you are pregnant. You may need to have a negative pregnancy test before starting this treatment. Use effective birth control to prevent pregnancy while you are using this medicine and for at least 6 months after your last dose.
• If you are a man, use effective birth control if your sex partner is able to get pregnant. Keep using birth control for at least 3 months after your last dose.
• Tell your doctor right away if a pregnancy occurs while either the mother or the father is using lorlatinib.

This medicine may affect fertility (ability to have children) in men. However, it is important to use birth control to prevent pregnancy because lorlatinib can harm an unborn baby.

Lorlatinib can make hormonal birth control less effective (birth control pills, injections, implants, skin patches, vaginal rings). Ask your doctor about the best form of birth control to use during treatment with this medicine.

Do not breastfeed while using this medicine, and for at least 7 days after your last dose.

How should I take lorlatinib?

Take lorlatinib exactly as prescribed by your doctor. Follow all directions on your prescription label and read all medication guides or instruction sheets.

Swallow the tablet whole and do not crush, chew, or break it.

Take the medicine at the same time each day, with or without food.

Do not use a broken or cracked tablet.

If you vomit shortly after taking lorlatinib, do not take another dose. Wait until your next scheduled dose time to take the medicine again.

Store at room temperature away from moisture and heat.

Do not change your dose or stop taking lorlatinib without your doctor’s advice.

Dosing information

Usual Adult Dose for Non-Small Cell Lung Cancer:

100 mg orally once a day until disease progression or unacceptable toxicity

Use: For the treatment of patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) whose disease has progressed on crizotinib and at least one other ALK inhibitor for metastatic disease OR alectinib as the first ALK inhibitor therapy for metastatic disease
OR ceritinib as the first ALK inhibitor therapy for metastatic disease.

What happens if I miss a dose?

Take the medicine as soon as you can, but skip the missed dose if your next dose is due in less than 4 hours. Do not use two doses at one time.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line .

What should I avoid while taking lorlatinib?

Avoid taking an herbal supplement containing St. John’s wort.

Lorlatinib side effects

Get emergency medical help if you have signs of an allergic reaction to lorlatinib: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Call your doctor at once if you have:

• sudden chest pain, wheezing, dry cough;
• shortness of breath or rapid breathing;
• fever, chills, cough with mucus;
• sudden dizziness (like you might pass out);
• numbness, tingling, or burning pain in your hands or feet;
• unusual changes in mood or behavior, thoughts of hurting yourself;
• hallucinations; or
• problems with speech, thought, or memory.

Your cancer treatments may be delayed or permanently discontinued if you have certain side effects.

Common lorlatinib side effects may include:

• swelling in your arms, hands, legs, or feet;
• numbness, tingling, or burning pain in your hands or feet;
• mood changes;
• feeling tired;
• weight gain;
• joint pain; or
• diarrhea.

What is Ofev?

Ofev (nintedanib) is used to treat a lung disease called idiopathic pulmonary fibrosis (IPF), interstitial lung disease when lung fibrosis is worsening, and systemic sclerosis-associated interstitial lung disease (SSc-ILD) to help slow the decline in lung function. Ofev capsules are taken twice daily.

Ofev is used in people with diseases that cause scar tissue (fibrosis) to form deep within the lungs. The scar tissue thickens and becomes stiff over time, making it harder for the lungs to work. Decreased lung function can make it hard to breathe. Other medical problems can occur when the brain, heart, and other organs do not get enough oxygen.

Ofev is not a cure for IPF or ILD, but this medicine can slow the effects of these diseases on your lungs.

Ofev mechanism of action is an inhibitor of multiple receptor tyrosine kinases (RTKs) and non-receptor tyrosine kinases (nRTKs). Some of these kinases are involved in fibrotic tissue remodeling in interstitial lung diseases, so blocking the kinases helps slow lung disease progression.

Is Ofev chemotherapy? No, Ofev is not chemotherapy. Another brand of nintedanib called Vargatef is used for cancer and is a targeted therapy.

Ofev side effects

Common side effects

Common Ofev side effects may include:

• nausea, vomiting, loss of appetite;
• stomach pain;
• diarrhea, weight loss;
• increased blood pressure;
• headache; or
• abnormal liver function tests.

Serious Ofev side effects 

Get emergency medical help if you have signs of an allergic reaction to Ofev with symptoms of hives, difficulty breathing, and swelling of your face, lips, tongue, or throat.

Call your doctor at once if you have:

• severe ongoing nausea, vomiting, or diarrhea;
• severe stomach pain, bloating, or tenderness;
• bleeding from your rectum or blood in your stools;
• easy bruising or bleeding, any wound that will not heal;
• fever, chills, cough with mucus, chest pain, feeling short of breath;
• heart attack symptoms – chest pain or pressure, pain spreading to your jaw or shoulder, nausea, sweating;
• liver problems – stomach pain (upper right side), loss of appetite, tiredness, dark urine, jaundice (yellowing of the skin or eyes); or
• signs of a stroke – sudden numbness or weakness (especially on one side of the body), slurred speech, problems with vision or balance.

Liver problems may be more likely in women, in people who weigh less than 143 pounds (65 kilograms), and in people of Asian descent.

Your doses may be delayed if you have certain side effects.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects.

Warnings

Ofev can cause birth defects or death in an unborn baby. Avoid getting pregnant while you are using this medicine and for at least 3 months after your last dose. Use effective birth control, with an extra barrier method (condom, diaphragm, cervical cap, contraceptive sponge).

Before taking this medicine

To make sure Ofev is safe for you, tell your doctor if you have ever had:

• liver disease;
• heart problems;
• a blood clot;
• bleeding problems (in you or a family member);
• surgery on your stomach or intestines;
• diverticulitis;
• if you smoke; or
• if you take a blood thinner such as warfarin (Coumadin, Jantoven).

You may need to have a negative pregnancy test before starting this treatment.

Nintedanib can cause birth defects or death in an unborn a baby. Use effective birth control to prevent pregnancy while you are using this medicine and for at least 3 months after your last dose. Tell your doctor if you become pregnant.

If you use hormonal birth control (birth control pills, injections, implants, skin patches, or vaginal rings), also use a barrier form of birth control to prevent pregnancy while taking Ofev. Barrier forms include a condom, diaphragm, cervical cap, or contraceptive sponge).

Ofev may affect fertility (your ability to have children) in women. However, it is important to use birth control to prevent pregnancy because this medicine can harm an unborn baby.

You should not breastfeed while using this medicine.

Ofev is not approved for use by anyone younger than 18 years old.

How should I take Ofev?

Ofev is usually taken 2 times per day, every 12 hours. Follow all directions on your prescription label and read all medication guides or instruction sheets. Your doctor may occasionally change your dose.

Your doctor will perform blood tests to make sure you do not have conditions that would prevent you from safely using Ofev.

Take with food if you have no liver impairment and apart from food if you have mild hepatic impairment. Take capsules with a full glass of water.

Swallow the capsule whole and do not crush, chew, break, or open it.

Take your doses at regular intervals, usually every 12 hours.

Ofev can cause severe diarrhea, which can be life-threatening if it leads to dehydration. You may need to take medication to prevent or quickly treat diarrhea.

Your doctor may recommend you have an anti-diarrhea medicine such as loperamide (Imodium) available at all times while you are taking Ofev. Take the anti-diarrhea medicine as directed on the label, or as prescribed by your doctor.

Drink plenty of liquids and call your doctor at once if you have diarrhea while taking Ofev.

You will need frequent medical tests to be sure nintedanib is not causing harmful effects on your liver. Your next few doses may be delayed based on the results.

Store this medicine in the original container at room temperature, away from moisture and heat.

Ofev Dosing information

Usual Adult Dose (no hepatic impairment)

Recommended Ofev dose 150 mg orally every 12 hours apart from food. Maximum dose: 300 mg/day.

Usual Adult Dose (mild hepatic impairment)

Recommended dosage in patients with mild hepatic impairment (Child-Pugh A): 100 mg taken orally twice daily, approximately 12 hours taken with food. 

Ofev is not recommended for use in patients with moderate or severe hepatic impairment.

General Ofev dosing information

• Do not chew, crush, or open capsules. If you or your caregiver accidentally comes in contact with the content of the capsule, wash your hands well right away.
• Consider temporary dose reduction to 100 mg, treatment interruption, or discontinuation for management of adverse reactions.
• Conduct liver function tests before starting therapy.
• Conduct a pregnancy test in females of reproductive potential before starting therapy.

What happens if I miss a dose?

Skip the missed dose and use your next dose at the regular time. Do not use two doses at one time.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line .

Do not take more than 300 milligrams of nintedanib in one day.

What to avoid

Smoking can make Ofev less effective. Avoid smoking while taking this medicine, or try to quit before you start this treatment.

Avoid taking an herbal supplement containing St. John’s wort.

Ask your doctor before taking a nonsteroidal anti-inflammatory drug (NSAID) such as aspirin, ibuprofen (Advil, Motrin), naproxen (Aleve), celecoxib (Celebrex), diclofenac, indomethacin, meloxicam, and others. Using an NSAID with nintedanib may cause you to bruise or bleed easily.

What other drugs will affect Ofev?

Sometimes it is not safe to use certain medications at the same time. Some drugs can affect your blood levels of other drugs you take, which may increase side effects or make the medications less effective.

Other drugs may interact with nintedanib, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.

What is Ojjaara?

Ojjaara (momelotinib) may be used to treat adults with certain types of myelofibrosis (MF) who have anemia. 

Myelofibrosis is a rare blood cancer where the bone marrow becomes scarred, preventing it from producing enough healthy blood cells. Over time, abnormal blood cells build up and replace normal ones. The exact cause is unknown, but it’s linked to a problem with a specific cell signaling pathway, called the JAK-STAT pathway. Treatment usually involves managing symptoms such as anemia and an enlarged spleen.

Ojjaara’s mechanism of action involves targeting and blocking specific enzymes called JAK1, JAK2, and a mutated form of JAK2. These enzymes are involved in regulating blood cell production and inflammation. Mutations in these enzymes have been associated with certain blood cancers like several leukemias, lymphomas, and myeloproliferative diseases. Ojjaara is not chemotherapy, it is a targeted treatment. 

Ojjaara gained FDA approval on September 15, 2023. There is no Ojjaara generic. Ojjaara belongs to the drug class known as multikinase inhibitors; specifically, it is a Janus Kinase inhibitor.

Ojjaara side effects

The most common side effects of Ojjaara include:

• low platelet counts
• dizziness
• bleeding
• diarrhea
• bacterial infections
• nausea
• tiredness.

Serious side effects and warnings

Ojjaara may cause the following serious side effects.

Serious and fatal infections. People who take Ojjaara may develop serious infections that can lead to death, such as bacterial and viral infections, including COVID-19. If you have an active infection, your healthcare provider should not start treatment with Ojjaara until your infection is gone. If you have had hepatitis B for a long time (chronic), Ojjaara may cause your hepatitis B to become active again, and your healthcare provider will check your blood for active hepatitis B before starting treatment. Your healthcare provider will monitor you and treat you for any infections that you get during treatment with Ojjaara. Tell your healthcare provider right away if you develop any of the following symptoms of infection:

• fever
• diarrhea
• chills
• vomiting
• cough
• pain or burning feeling when passing urine
• breathing problems.

Low platelet and white blood cell counts. Ojjaara may cause new or worsening low platelet and white blood cell counts. Low platelet counts may increase your risk for bleeding and low white blood cell counts may increase your risk for infection. Your healthcare provider will do blood tests to check your blood counts before you start taking Ojjaara, and during treatment. Tell your healthcare provider right away if you have any signs of bleeding during treatment with Ojjaara, including:

• unusual bleeding
• bruising
• black or tarry stools.

Liver problems. Ojjaara may cause new or worsening increased liver enzymes and bilirubin in your blood. Your healthcare provider will check your liver enzymes before starting treatment, every month for the first 6 months of treatment, and then as needed during treatment with Ojjaara. Your healthcare provider may stop treatment with Ojjaara if your liver enzymes increase. Tell your healthcare provider if you develop any of the following signs or symptoms of liver problems:

• tiredness
• dark urine
• loss of appetite
• yellowing of your skin or the white part of your eyes
• pain in your right upper stomach area (abdomen).

Major cardiovascular events such as heart attack, stroke, and death. Major cardiac events have happened, especially in people with cardiac risk factors and who are current or past smokers, taking another Janus kinase (JAK) inhibitor to treat rheumatoid arthritis. Ojjaara is in the JAK family of medicines. Get emergency help right away if you have any symptoms of a heart attack or stroke while taking Ojjaara, including:

• discomfort in your chest that lasts for more than a few minutes, or that goes away and comes back
• severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw
• pain or discomfort in your arms, back, neck, jaw, or stomach
• shortness of breath with or without chest discomfort
• breaking out in a cold sweat
• nausea or vomiting
• feeling lightheaded
• weakness in one part or on one side of your body
• slurred speech.

Blood clots. Blood clots in the veins of the legs (deep vein thrombosis, DVT) or lungs (pulmonary embolism, PE) have happened in some people taking another JAK inhibitor to treat rheumatoid arthritis, and may be life-threatening. Tell your healthcare provider if you have had blood clots in the veins of your legs or lungs in the past. Tell your healthcare provider right away if you have any signs and symptoms of blood clots during treatment with Ojjaara, including:

• swelling, pain, or tenderness in one or both legs
• sudden, unexplained chest pain
• shortness of breath or difficulty breathing.

New cancers. New cancers, including lymphoma and other cancers, except non-melanoma skin cancer, have happened in some people taking another JAK inhibitor to treat rheumatoid arthritis. The risk of new cancers is further increased in people who smoke or who smoked in the past.

It is not known if Ojjaara is safe and effective for children.

These are not all of the possible side effects of Ojjaara. Call your doctor for medical advice about side effects.

Before taking

Do not take Ojjaara if you are allergic to momelotinib, Ojjaara, or any of the inactive ingredients in the Ojjaara tablets.

Taking Ojjaara can increase your risk of life-threatening medical problems, including serious infections, a heart attack or stroke, blood clots, or cancer. Tell your doctor if you’ve had:

• a weak immune system or chronic infection (such as tuberculosis, shingles, HIV, or hepatitis B)
• any type of cancer
• chronic lung disease
• heart problems
• a heart attack, stroke, or blood clot
• liver disease
• if you’ve ever smoked
• if you are scheduled to receive any vaccine.

Before taking Ojjaara, tell your healthcare provider about all of your medical conditions, including if
you:

• have an infection or have any of the following symptoms: fever, chills, muscle aches, cough, trouble breathing, skin sores, diarrhea, or painful urination
• have or have had hepatitis B
• have or have had liver problems
• have had a heart attack, or have or have had other heart problems, or stroke
• have or have had a blood clot
• smoke or were a smoker in the past
• have or have had any other cancers
• are pregnant or plan to become pregnant
• are breastfeeding or plan to breastfeed.

Pregnancy

Ojjaara may harm your unborn baby. You should use effective birth control (contraception) during treatment and for 1 week after the last dose of Ojjaara. Tell your healthcare provider right away if you think you are pregnant or become pregnant during treatment.

Breastfeeding

It is not known if Ojjaara passes into your breast milk. You should not breastfeed during treatment and for 1 week after the last dose of Ojjaara. Talk to your healthcare provider about the best way to feed your baby during this time

How should I take Ojjaara?

Take Ojjaara exactly as your healthcare provider tells you to take it. Do not change your dose or stop taking Ojjaara without first talking to your healthcare provider. Your healthcare provider may change your dose, temporarily stop, or permanently stop treatment if you have certain side effects.

• Ojjaara is a tablet that is taken by mouth once a day.
• Ojjaara tablets may be taken with or without food.
• Swallow the tablets whole. Do not cut, crush, or chew tablets.
• Your healthcare provider will do blood tests before you start taking Ojjaara and during treatment.

Ojjaara dosing information

• The usual dosage is Ojjaara 200mg once daily.
• In people with severe liver disease (Child-Pugh Class C), your healthcare provider may reduce your dose.

What happens if I miss a dose?

Take the missed dose as soon as you remember. Skip the missed dose and take your next dose the following day at your regularly scheduled time. Do not take 2 doses at the same time to make up for the missed dose.

What happens if I overdose?

If you take too much Ojjaara, call your healthcare provider or go to the nearest emergency room right away and take your bottle of Ojjaara with you.

What other drugs will affect Ojjaara?

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking Ojjaara with certain other medicines may affect the amount of it or the other medicines in your blood and may increase your risk of side effects. Tell your doctor if you take:

• Organic Anion Transporting Polypeptide (OATP)1B1/B3 inhibitors, such as atazanavir, clarithromycin, cobicistat, cyclosporine
• Breast Cancer Resistance Protein (BCRP) substrates, such as prazosin, sulfasalazine, and rosuvastatin

Know the medicines you take. Keep a list of the medicines you take to show your healthcare provider and pharmacist when you get a new medicine.

Storage

Store Ojjaara at room temperature between 68°F to 77°F (20°C to 25°C).

Keep it in its original bottle. The Ojjaara bottle contains a desiccant packet to help keep your tablets dry (protect from moisture). Keep the desiccant in the bottle. Tightly close the bottle after you take your dose.

Keep out of the reach of children.

Ojjaara ingredients

The ingredients of Ojjaara made by GSK are as follows:

Active ingredient: momelotinib dihydrochloride monohydrate

Inactive ingredients: lactose monohydrate, magnesium stearate, microcrystalline cellulose, propyl gallate, silicon dioxide, and sodium starch glycolate. Film coating: polyethylene glycol, polyvinyl alcohol, red iron oxide, talc, titanium dioxide, and yellow iron oxide.

Available as Ojjaara 100 mg tablets, Ojjaara 150 mg tablets, and Ojjaara 200 mg tablets.

What is Qinlock?

Qinlock is an enzyme inhibitor that blocks the action of protein kinases.

Qinlock is a prescription medicine used to treat tumors of the stomach and intestines, specifically adults with advanced gastrointestinal stromal tumor (GIST).

Qinlock is for use in adults who have already been treated with at least 3 other cancer medicines.

Warnings

Qinlock may affect fertility in males which may affect the ability to have children. Talk to your healthcare provider if this is a concern for you.

Qinlock and certain other medicines can affect each other causing side effects or affect how ripretinib works. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Follow all directions on your medicine label and package. Tell each of your healthcare providers about all your medical conditions, allergies, and all medicines you use.

Before taking this medicine

You may not be able to use Qinlock if you have untreated or uncontrolled hypertension (high blood pressure).

To make sure Qinlock is safe for you, tell your doctor if you have ever had:

• skin problem called palmar-plantar erythrodysesthesia syndrome;
• high blood pressure;
• heart problems; or
• if you have recently had surgery or plan to have surgery.

Using Qinlock may increase your risk of developing skin cancer. Your skin may need to be checked while you are using this medicine. Ask your doctor about skin symptoms to watch for.

You may need to have a negative pregnancy test before starting this treatment.

Both men and women using this medicine should use effective birth control to prevent pregnancy. Ripretinib can harm an unborn baby if the mother or father is using this medicine.

Use effective birth control to prevent pregnancy while you are using this medicine and for at least 1 week after your last dose. Tell your doctor right away if a pregnancy occurs while either the mother or the father is using Qinlock.

This medicine may affect fertility (ability to have children) in men. However, it is important to use birth control to prevent pregnancy because ripretinib can harm an unborn baby.

Do not breastfeed while using this medicine, and for at least 1 week after your last dose.

How should I take Qinlock?

Take Qinlock exactly as prescribed by your doctor. Follow all directions on your prescription label and read all medication guides or instruction sheets. Your doctor may occasionally change your dose.

Swallow the tablet whole and do not crush, chew, or break it.

Take the medicine at the same time each day, with or without food.

If you vomit shortly after taking Qinlock, do not take another dose. Wait until your next scheduled dose time to take the medicine again.

Your blood pressure will need to be checked often. Your heart function may also need to be checked.

If you need surgery, tell your surgeon you currently use this medicine. You may need to stop for a short time.

Your cancer treatments may be delayed or permanently discontinued if you have certain side effects.

Do not change your dose or stop taking Qinlock without your doctor’s advice.

Store at room temperature away from moisture and heat. Keep the tablets in their original container, along with the packet or canister of moisture-absorbing preservative.

Dosing information

Usual Adult Dose for Gastrointestinal Stromal Tumor:

150 mg orally once daily until disease progression or unacceptable toxicity

Use: For adults with advanced gastrointestinal stromal tumor (GIST) who have received prior treatment with 3 or more kinase inhibitors, including imatinib

What happens if I miss a dose?

Take the medicine as soon as you can, but skip the missed dose if you are more than 8 hours late for the dose. Do not take two doses at one time.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line .

What to avoid

Qinlock could make you sunburn more easily. Avoid sunlight or tanning beds. Wear protective clothing and use sunscreen (SPF 30 or higher) when you are outdoors.

Qinlock side effects

Get emergency medical help if you have signs of an allergic reaction to Qinlock: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Call your doctor at once if you have:

• severe stomach pain, nausea, or vomiting;
• severe headache, blurred vision, pounding in your neck or ears;
• pain, blisters, bleeding, or severe rash in the palms of your hands or the soles of your feet;
• heart problems – tiredness, feeling short of breath, swollen veins in the sides of your neck, swelling in your stomach or lower legs;
• low red blood cells (anemia) – pale skin, unusual tiredness, feeling light-headed or short of breath, cold hands and feet; or
• skin changes – a new wart, a skin sore or red bump that bleeds or does not heal, or any change in the size or color of a mole.

Common Qinlock side effects may include:

• nausea, vomiting, stomach pain, loss of appetite;
• diarrhea, constipation;
• tiredness;
• muscle pain; or
• thinning hair, hair loss.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects.

What other drugs will affect Qinlock?

Sometimes it is not safe to use certain medications at the same time. Some drugs can affect your blood levels of other drugs you take, which may increase side effects or make the medications less effective.

Tell your doctor about all your other medicines, especially blood pressure medicine.

Other drugs may interact with ripretinib, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.

What is quizartinib?

Quizartinib is used in adults alone or in combination with other cancer treatments to treat acute myeloid leukemia.

Quizartinib is available only from a certified pharmacy under a special program. You must be registered in the program and understand the risks and benefits of quizartinib.

Quizartinib may also be used for purposes not listed in this medication guide.

Quizartinib side effects

Get emergency medical help if you have signs of an allergic reaction: hives, difficult breathing, swelling of your face, lips, tongue, or throat.

Quizartinib can cause a serious heart problem, especially if you use certain medicines at the same time. Call your doctor right away if you have irregular heartbeat, a light-headed feeling, like you might pass out, or have diarrhea or vomiting.

Your dose may be decreased and your treatment may be delayed or permanently discontinued if you have certain side effects.

Common side effects of quizartinib may include:

• nausea, vomiting, stomach pain, upset stomach, diarrhea, loss of appetite;
• fungal infections;
• sepsis (a severe reaction to an infection);
• eye irritation;
• nosebleeds;
• cold symptoms such as stuffy nose, sneezing, sore throat;
• herpes infection;
• trouble sleeping;
• low levels of platelets in your blood;
• an electrolyte imbalance (such as low blood levels of potassium or magnesium);
• changes in liver function tests;
• an abnormal electrocardiogram or ECG (sometimes called an EKG); or
• low blood cell counts–fever, chills, tiredness, mouth sores, skin sores, easy bruising, unusual bleeding, pale skin, cold hands and feet, feeling light-headed or short of breath.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects.

Warnings

Quizartinib can cause a serious heart problem, especially if you use certain medicines at the same time. Call your doctor right away if you have irregular heartbeat, a light-headed feeling, like you might pass out, or have diarrhea or vomiting.

Quizartinib is available only from a certified pharmacy under a special program. You must be registered in the program and understand the risks and benefits of quizartinib.

Before taking this medicine

You should not use quizartinib if you are allergic to it, or if you have:

• very low magnesium level;
• very low potassium level;
• long QT syndrome; or
• history of ventricular arrhythmias or torsades de pointes.

Tell your doctor if you have or have ever had:

• heart problems; or
• low blood levels of potassium or magnesium.

Quizartinib can harm an unborn baby if the mother or the father is using this medicine.

• If you are a woman, you will need a pregnancy test to make sure you are not pregnant. Use birth control while using this medicine and for at least 7 months after your last dose.
• If you are a man, use birth control if your sex partner is able to get pregnant. Keep using birth control for at least 4 months after your last dose.
• Tell your doctor right away if a pregnancy occurs.

Pregnancy may be less likely to occur while the mother or the father is using this medicine. Both men and women should still use birth control to prevent pregnancy because the medicine can harm an unborn baby.

Do not breastfeed while using this medicine, and for at least one month after your last dose.

How should I take quizartinib?

Follow all directions on your prescription label and read all medication guides or instruction sheets. Use the medicine exactly as directed.

Do not change your dose or stop taking the medicine without your doctor’s advice.

Quizartinib is usually taken once per day with or without food at the same time each day.

If you vomit shortly after taking quizartinib, do not take another dose. Take your next dose as scheduled.

Swallow the tablet whole and do not crush, chew, or break it.

You may need medical tests before and during your treatment with quizartinib to check your heart function using an electrocardiograph or ECG (sometimes called an EKG).

Your blood will be tested before and during treatment with quizartinib.

Store at room temperature away from moisture and heat.

What happens if I miss a dose?

Take the medicine as soon as you can, but skip the missed dose if it is almost time for your next dose. Do not take two doses at one time.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line .

What should I avoid while taking quizartinib?

Avoid taking an herbal supplement containing St. John’s wort.

What other drugs will affect quizartinib?

Sometimes it is not safe to use certain medicines at the same time. Some drugs can affect your blood levels of other drugs you use, which may increase side effects or make the medicines less effective.

What is Retevmo?

Retevmo (selpercatinib) is an oral kinase inhibitor that may be used to treat the following cancers caused by abnormal RET (rearranged during transfection) genes:

• locally advanced non-small cell lung cancer (NSCLC) or NSCLC that has spread in adults
• advanced or metastatic thyroid cancer in adults and children aged 2 years of age and older who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate)
• advanced medullary thyroid cancer (MTC) or MTC that has spread and requires a medicine that treats the whole body (systemic therapy) in adults and children aged 2 years and older.

Retevmo is also approved under the accelerated approval designation for adults and children aged 2 years and older with locally advanced solid tumors (cancers) or solid tumors associated with abnormal RET genes that have spread or gotten worse (progressed) after other treatments, or who have no satisfactory alternative treatment options.

Note: Continued approval under the accelerated approval designation is contingent upon a continued benefit in clinical trials.

Your healthcare provider will perform a test for abnormal RET genes to make sure that Retevmo is right for you.

It is not known if Retevmo is safe and effective when used in children younger than 2 years of age for the treatment of MTC, thyroid cancer, solid tumors, or other cancers.

Retevmo first gained FDA-approval on May 8, 2020.

Retevmo side effects

The most common side effects of Retevmo include:

• swelling of your arms, legs, hands, and feet (edema)
• diarrhea
• tiredness
• dry mouth
• high blood pressure
• stomach-area (abdominal) pain
• constipation
• rash
• nausea
• headache.

The most common severe abnormal laboratory test results with Retevmo include decreased white blood cell count, decreased levels of sodium in the blood, and decreased levels of calcium in the blood.

Retevmo may affect fertility in females and males, which may affect your ability to have children. Talk to your healthcare provider if this is a concern for you.

Serious side effects

Retevmo may cause serious side effects, including:

• Liver problems. Liver problems (increased liver enzymes) are common with Retevmo and may sometimes be serious. Your healthcare provider will do blood tests before and during treatment to check for liver problems. Tell your healthcare provider right away if you get any of the following symptoms of liver problems during treatment:
  • yellowing of your skin or the white part of your eyes (jaundice)
  • dark “tea-colored” urine
  • sleepiness
  • bleeding or bruising
  • loss of appetite
  • nausea or vomiting
  • pain on the upper right side of your stomach area
• Lung problems. Retevmo may cause severe or life-threatening inflammation of the lungs during treatment, that can lead to death. Tell your healthcare provider right away if you have any new or worsening lung symptoms, including:
  • shortness of breath
  • cough
  • fever
• High blood pressure (hypertension). High blood pressure is common with Retevmo and may sometimes be serious. You should check your blood pressure regularly during treatment. Tell your healthcare provider if you get any of the following symptoms:
  • confusion
  • headaches
  • shortness of breath
  • dizziness
  • chest pain
• Heart rhythm changes (QT prolongation) can occur and may be serious. Retevmo may cause very slow, very fast or irregular heartbeats. Tell your healthcare provider right away if you get any of the following symptoms:
  • loss of consciousness
  • fainting
  • dizziness
  • a change in the way your heart beats (heart palpitations)
• Bleeding problems. Retevmo can cause bleeding which can be serious and may lead to death. Tell your healthcare provider if you have any signs of bleeding during treatment, including:
  • vomiting blood or if your vomit looks like coffee-grounds
  • pink or brown urine
  • red or black (looks like tar) stools
  • coughing up blood or blood clots
  • unusual bleeding or bruising of your skin
  • menstrual bleeding that is heavier than normal
  • unusual vaginal bleeding
  • nose bleeds that happen often
  • drowsiness or difficulty being awakened
  • confusion
  • headache
  • change in speech
• Allergic reactions. Retevmo can cause a fever, rash, muscle or joint pain, especially in the first month of treatment. Tell your healthcare provider if you get any of these symptoms.
• Tumor lysis syndrome (TLS). TLS is caused by a fast breakdown of cancer cells. TLS can cause kidney failure, the need for dialysis treatment, and an abnormal heartbeat. TLS can lead to hospitalization. Your healthcare provider may do blood tests to check you for TLS. You should stay well hydrated during treatment with Retevmo. Call your healthcare provider or get emergency medical help right away if you develop any of these symptoms during treatment with Retevmo:
  • nausea
  • shortness of breath
  • vomiting
  • muscle cramps
  • weakness
  • seizures
  • swelling
• Risk of wound healing problems. Wounds may not heal properly during treatment with Retevmo. Tell your healthcare provider if you plan to have any surgery before or during treatment with Retevmo.
  • Stop taking Retevmo at least 7 days before planned surgery.
  • Your healthcare provider should tell you when you may start taking Retevmo again after surgery.

A hip condition called a slipped capital femoral epiphysis/slipped upper femoral epiphysis (SCFE/SUFE) that can occur in teens who are still growing was reported in one adolescent in clinical trials. Report any pain in the groin, hip, knee, or thigh to your healthcare provider especially if it is intermittent or worsens with activity.

Before taking Retevmo

Before you start treatment, tell your healthcare provider about all your medical conditions, including if you:

• have liver problems
• have high blood pressure
• have heart problems including a condition called QT prolongation
• have bleeding problems
• plan to have surgery. Stop taking Retevmo at least 7 days before your planned surgery. See Retevmo side effects.
• are pregnant or plan to become pregnant. Retevmo can harm your unborn baby. You should not become pregnant during treatment.
  • If you are able to become pregnant, your healthcare provider will do a pregnancy test before you start treatment.
  • Females who are able to become pregnant should use effective birth control (contraception) during treatment and for at least 1 week after the final dose. Talk to your healthcare provider about birth control methods that may be right for you.
  • Tell your healthcare provider right away if you become pregnant or think you might be pregnant during treatment.
  • Males with female partners who are able to become pregnant should use effective birth control during treatment, and for at least 1 week after the final dose.
• are breastfeeding or plan to breastfeed. It is not known if Retevmo passes into your breast milk. Do not breastfeed during treatment, and for 1 week after the last dose.

How should I take Retevmo?

Take Retevmo exactly as your healthcare provider tells you. Your healthcare provider may stop treatment or change your dose if you have side effects. Do not change your dose or stop treatment unless your healthcare provider tells you.

• Retevmo is taken by mouth, usually 2 times a day (about 12 hours apart) with or without food.
• If you take a proton-pump inhibitor (PPIs such as dexlansoprazole, esomeprazole, lansoprazole, omeprazole, pantoprazole sodium, and rabeprazole), take Retevmo with food.
• If you take an antacid that contains aluminum, magnesium, calcium, simethicone, or buffered medicines, take Retevmo 2 hours before or 2 hours after taking the antacid.
• If you take an H2 blocker (such as famotidine, nizatidine, and cimetidine), take Retevmo 2 hours before or 10 hours after taking the H2 blocker.

Swallow Retevmo capsules whole. Do not chew or crush the tablets.

• Do not give Retevmo capsules to your child if they cannot swallow a capsule. 

If you vomit after taking a dose, do not take an extra dose. Take the next dose at your scheduled time.

• Do not take a missed dose unless it is more than 6 hours until your next scheduled dose.
• If you take too much Retevmo, call your healthcare provider or go to the nearest hospital emergency room right away.

Dosing information

Usual Retevmo dose for adults and adolescents over the age of 12

• Less than 50 kg: 120 mg, 2 times daily (approximately every 12 hours).
• 50 kg or greater: 160 mg, 2 times daily (approximately every 12 hours).

Usual Retevmo dose for children aged 2 years to less than 12 years

Dosage is based on body surface area.

• 0.33 to 0.65 m²: 40 mg, 3 times daily
• 0.66 to 1.08 m²: 80 mg, 2 times daily
• 1.09 to 1.52 m²: 120 mg, 2 times daily
• At least 1.53 m²: 160 mg, 2 times daily.

What other drugs will affect Retevmo?

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Certain other medicines may affect how Retevmo works.

You should avoid taking the following medicines during treatment with Retevmo:

• St. John’s wort
• proton pump inhibitors (PPIs such as dexlansoprazole, esomeprazole, lansoprazole, omeprazole, pantoprazole sodium, rabeprazole)
• H2 blockers (such as famotidine, nizatidine, and cimetidine), and
• antacids that contain aluminum, magnesium, calcium, simethicone, or buffered medicines.

If you cannot avoid taking PPIs, H2 blockers, or antacids, see How should I take Retevmo? for more information on how to take Retevmo with these medicines.

Know the medicines you take. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.

How should I store Retevmo?

• Store Retevmo capsules at room temperature between 68˚F to 77˚F (20˚C to 25˚C).
• Keep all medicines out of the reach of children and pets.

What are the ingredients in Retevmo?

Active ingredient: selpercatinib

Inactive ingredients (capsules): colloidal silicon dioxide and microcrystalline cellulose.

• 40 mg capsule shell: gelatin, titanium dioxide, ferric oxide black and black ink.
• 80 mg capsule shell: gelatin, titanium dioxide, FD&C blue #1 and black ink.
• Black ink: shellac, potassium hydroxide, and ferric oxide black.

Inactive ingredients (tablets): croscarmellose sodium, hydroxypropyl cellulose, mannitol, microcrystalline cellulose, and sodium stearyl fumarate.

• Tablet film coating: polyvinyl alcohol, titanium dioxide, polyethylene glycol, and talc.
• Film coating of 40 mg, 80 mg, and 120 mg tablets: ferrosoferric oxide.
• Film coating of 80 mg, 120 mg, and 160 mg tablets: ferric oxide. 

Available as:

• Capsules: 40mg, 80mg
• Tablets: 40mg, 80mg, 120mg, 160mg.

What is Rydapt?

Rydapt is a cancer medicine that interferes with the growth and spread of cancer cells in the body.

Rydapt is used together with other cancer medicines to treat acute myeloid leukemia.

Rydapt is also used to treat certain rare blood disorders, including systemic mastocytosis with mast cell leukemia or other cancers affecting the blood, bone marrow, or lymphatic tissue.

Warnings

Both men and women using Rydapt should use effective birth control to prevent pregnancy. Midostaurin can harm an unborn baby if the mother or father is using this medicine.

Before taking this medicine

You should not use Rydapt if you are allergic to midostaurin.

Tell your doctor if you have ever had:

• lung disease or breathing problems.

You may need to have a negative pregnancy test before starting this treatment.

Midostaurin can harm an unborn baby or cause birth defects if the mother or the father is using Rydapt.

• If you are a woman, do not use midostaurin if you are pregnant. Use effective birth control to prevent pregnancy while you are using this medicine and for at least 4 months after your last dose. If you are pregnant, your name may be listed on a pregnancy registry to track the effects of midostaurin on the baby.
• If you are a man, use effective birth control if your sex partner is able to get pregnant. Keep using birth control for at least 4 months after your last dose.
• Tell your doctor right away if a pregnancy occurs while either the mother or the father is using Rydapt.

Rydapt may affect fertility (ability to have children) in both men and women. However, it is important to use birth control to prevent pregnancy because midostaurin can harm an unborn baby.

You should not breastfeed while using Rydapt and for at least 4 months after your last dose.

Rydapt is not approved for use by anyone younger than 18 years old.

How should I take Rydapt?

Take Rydapt exactly as prescribed by your doctor. Your doctor will perform a test to make sure Rydapt is the right treatment for your condition.

Follow all directions on your prescription label and read all medication guides or instruction sheets. Use the medicine exactly as directed.

Rydapt is usually taken 2 times per day (once every 12 hours) with food.

Take your doses at regular intervals to keep a steady amount of the drug in your body at all times.

You may be given medication to prevent nausea or vomiting while you are taking this medicine.

If you vomit shortly after taking Rydapt, do not take another dose. Wait until your next scheduled dose time to take the medicine again.

Do not crush or open a Rydapt capsule.

You may need frequent blood tests (every 1 to 4 weeks). Rydapt is usually given until your body no longer responds to the medication.

Use all medications as directed by your doctor. Read the instructions provided with each medication. Do not change your doses or medication schedule without your doctor’s advice.

Store the capsules in the original container at room temperature, away from moisture and heat.

Dosing information

Usual Adult Dose for Acute Myeloid Leukemia:

50 mg orally twice a day at approximately 12 hour intervals on Days 8 to 21 of each cycle of induction AND on Days 8 to 21 of each cycle of consolidation.

Comments:
-This drug is not indicated as a single-agent induction therapy for acute myeloid leukemia (AML) patients.
-Information on FDA-approved tests for the detection of FLT3 mutation in AML is available at: http://www.fda.gov/CompanionDiagnostics.

Use: In combination with standard cytarabine and daunorubicin induction and high-dose cytarabine consolidation, treatment of newly diagnosed AML who are FLT3 mutation-positive as detected by a FDA approved test.

Usual Adult Dose for Systemic Mastocytosis:

100 mg orally twice a day at approximately 12 hour intervals
Duration of Therapy: Until disease progression or unacceptable toxicity occurs.

Comments: Monitor for toxicity at least weekly for the first 4 weeks, every other week for the next 8 weeks, and monthly thereafter while on treatment.

Uses: Treatment of aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL).

Usual Adult Dose for Leukemia:

100 mg orally twice a day at approximately 12 hour intervals
Duration of Therapy: Until disease progression or unacceptable toxicity occurs.

Comments: Monitor for toxicity at least weekly for the first 4 weeks, every other week for the next 8 weeks, and monthly thereafter while on treatment.

Uses: Treatment of aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL).

What happens if I miss a dose?

Take the medicine as soon as you can, but skip the missed dose if it is almost time for your next dose. Do not take two doses at one time.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line .

What to avoid

Follow your doctor’s instructions about any restrictions on food, beverages, or activity.

Rydapt side effects

Get emergency medical help if you have signs of an allergic reaction to Rydapt: hives, warmth, redness, or tingly feeling; chest pain, difficult breathing; swelling of your face, lips, tongue, or throat.

Call your doctor at once if you have:

• pain or sores in or around your mouth, redness inside your mouth, sore throat;
• sudden chest pain or discomfort, wheezing, dry cough, feeling short of breath;
• signs of infection around where a needle was injected to give you other medicines – bruising, swelling, warmth, redness, oozing, or bleeding around the needle;
• high blood sugar – increased thirst, increased urination, dry mouth, fruity breath odor; or
• low blood cell counts – fever, chills, tiredness, mouth sores, skin sores, easy bruising, unusual bleeding, pale skin, cold hands and feet, feeling light-headed or short of breath.

Common Rydapt side effects may include:

• low blood cell counts, fever;
• bruising, nosebleeds;
• high blood sugar;
• infection around a needle injection;
• mouth sores;
• cold symptoms such as stuffy nose, sneezing, sore throat;
• trouble breathing;
• nausea, vomiting, stomach pain, constipation, diarrhea;
• muscle or bone pain;
• headache, feeling tired; or
• swelling in your hands, feet, or ankles.

What is Alunbrig?

Alunbrig is a cancer medicine that interferes with the growth and spread of cancer cells in the body.

Alunbrig is used to treat non-small cell lung cancer that has spread to other parts of the body (metastatic).

Alunbrig is used only if your cancer has a specific genetic marker (an abnormal “ALK” gene). Your doctor will test you for this gene.

Warnings

Alunbrig can cause serious side effects such as lung problems, vision problems, high blood pressure, high blood sugar, very slow heartbeats, muscle damage, or pancreatitis.

Call your doctor at once if you have side effects, such as: severe headache, confusion, feeling light-headed, blurred vision, seeing flashes of light or “floaters” in your vision, increased thirst or urination, hunger, nausea, upper stomach pain spreading to your back, unexplained muscle pain or weakness, fever, cough, chest pain, trouble breathing, very slow heartbeats, or feeling like you might pass out.

Before taking this medicine

You should not use Alunbrig if you are allergic to brigatinib.

To make sure Alunbrig is safe for you, tell your doctor if you have ever had:

• lung disease or breathing problems;
• slow heartbeats;
• high blood pressure;
• vision problems;
• diabetes or glucose intolerance;
• pancreas problems;
• liver disease; or
• kidney disease (or if you are on dialysis).

You may need to have a negative pregnancy test before starting this treatment.

Brigatinib can harm an unborn baby if the mother or the father is using this medicine.

• If you are a woman, do not use this medicine if you are pregnant. Alunbrig can make hormonal birth control less effective, including birth control pills, injections, implants, skin patches, and vaginal rings. To prevent pregnancy while using Alunbrig, use an effective non-hormonal form of birth control such as an intrauterine device (IUD). Avoid getting pregnant for at least 4 months after your last dose.
• If you are a man, use condoms or other barrier birth control if your sex partner is able to get pregnant. Keep using condoms for at least 3 months after your last dose.
• Tell your doctor right away if a pregnancy occurs while either the mother or the father is using brigatinib.

Alunbrig may affect fertility (ability to have children) in men. However, it is important to use birth control to prevent pregnancy because brigatinib can harm an unborn baby.

You should not breastfeed while using this medicine, and for at least 1 week after your last dose.

How should I take Alunbrig?

Take Alunbrig exactly as prescribed by your doctor. Follow all directions on your prescription label and read all medication guides or instruction sheets. Your doctor may occasionally change your dose.

Alunbrig is usually taken once per day, with or without food.

Swallow the tablet whole and do not crush, chew, or break it.

If you vomit shortly after taking Alunbrig, do not take another dose. Stay on your regular schedule.

Your blood pressure will need to be checked often.

You should not stop using this medicine unless your doctor tells you to.

Store at room temperature away from moisture and heat.

Dosing information

Usual Adult Dose for Non-Small Cell Lung Cancer:

-Initial Dose: 90 mg orally once a day for the first 7 days
-Maintenance Dose: If initial dose is tolerated during the first 7 days, increase to 180 mg orally once a day.
-Duration of Therapy: Until disease progression or unacceptable toxicity

Use: Treatment of patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib.

What happens if I miss a dose?

Skip the missed dose and use your next dose at the regular time. Do not use two doses at one time.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line .

What to avoid

Grapefruit may interact with brigatinib and lead to unwanted side effects. Avoid the use of grapefruit products.

This medicine may cause blurred vision and may impair your reactions. Avoid driving or hazardous activity until you know how this medicine will affect you.

Alunbrig side effects

Get emergency medical help if you have signs of an allergic reaction to Alunbrig: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Call your doctor at once if you have:

• lung problems – cough, trouble breathing, chest pain, fever;
• vision problems – blurred vision, double vision, increased sensitivity to light, seeing flashes of light or “floaters” in your vision;
• high blood pressure – severe headache, pounding in your neck or ears, dizziness;
• high blood sugar – increased thirst, increased urination, hunger, nausea, fruity breath odor, weakness, confusion;
• heart problems – very slow heartbeats, feeling like you might pass out;
• muscle problems – unexplained muscle pain or weakness; or
• pancreatitis – upper stomach pain (worse with eating and may spread to your back), nausea, weight loss.

Your cancer treatments may be delayed or permanently discontinued if you have certain side effects.

Common Alunbrig side effects may include:

• nausea, vomiting, diarrhea;
• increased blood pressure;
• trouble breathing;
• cough;
• rash;
• muscle pain;
• headache; or
• feeling tired.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects.

What other drugs will affect Alunbrig?

Sometimes it is not safe to use certain medications at the same time. Some drugs can affect your blood levels of other drugs you take, which may increase side effects or make the medications less effective.

Many drugs can interact with brigatinib. This includes prescription and over-the-counter medicines, vitamins, and herbal products. Not all possible interactions are listed here. Tell your doctor about all your current medicines and any medicine you start or stop using.