6 mins read

Overview: Midostaurin is used to treat certain types of acute myeloid leukemia or systemic mastocytosis. Common side effects include nausea, vomiting, diarrhea, and headaches. Tell your health care provider if you have any of these that are severe or will not go away. Call your health care provider right away if you get a cough, fever, or trouble breathing. 

Uses

What is midostaurin used for?

Midostaurin is commonly used to treat certain types of the following cancers and rare diseases. 

Midostaurin may also be used for other conditions as determined by your health care provider.

How does midostaurin work (mechanism of action)?

If you take this medicine to treat cancer, your health care provider can run a test to see if your cancer may respond to midostaurin. Midostaurin is a type of medicine known as a tyrosine kinase inhibitor (TKI) that works by targeting specific receptors on certain cancer or mast cells. By blocking the action of these receptors, midostaurin can cause abnormal blood cancer cells to die. When used for systemic mastocytosis, midostaurin reduces the amount of mast cells. 

How is midostaurin supplied (dosage forms)?

Brands and other names

  • Rydapt

Dosage forms and strengths

  • 25 mg oral capsules

How should I store midostaurin?

Midostaurin should be stored at room temperature, between 68 F and 77 F (20 C and 25 C). It can be exposed to temperatures between 59 F and 86 F (15 C and 30 C) for shorter periods of time, such as when you're transporting it. Store it in a cool, dry place. Keep midostaurin in the original bottle.

Side Effects

What are the most common side effects of midostaurin?

The most common side effects of midostaurin are listed below. These side effects may vary, depending on the condition that is being treated. Tell your health care provider if you have any of these side effects that bother you.

  • Low white blood cell counts with fever (febrile neutropenia)
  • Nausea, vomiting, diarrhea, constipation, or stomach pain
  • Mouth sores
  • Headache
  • Bruising or nosebleeds
  • Muscle or bone pain
  • Swelling in your hands, feet, or ankles
  • Tiredness 
  • High blood sugar levels
  • Infection in the nose or throat, sore throat, or common cold symptoms
  • Abnormal heart rhythm (QT prolongation)

There may be other side effects of midostaurin that are not listed here. Contact your health care provider if you think you are having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088. In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.

What are the serious side effects of midostaurin?

While less common, the most serious side effects of midostaurin are described below, along with what to do if they happen.

Severe Allergic Reactions. Midostaurin may cause allergic reactions, which can be serious. Stop taking midostaurin and get help right away if you have any of the following symptoms of a serious allergic reaction.

  • Breathing problems or wheezing
  • Racing heart
  • Fever or general ill feeling
  • Swollen lymph nodes
  • Swelling of the face, lips, mouth, tongue, or throat
  • Trouble swallowing or throat tightness
  • Itching, skin rash, or pale red bumps on the skin called hives
  • Nausea or vomiting
  • Dizziness, feeling lightheaded, or fainting
  • Stomach cramps
  • Joint pain

Lung Inflammation. Midostaurin can cause inflammation in your lungs, also called interstitial lung disease or pneumonitis. This can be serious or even life-threatening. Get emergency help if you have trouble breathing. You may also have a cough, fever, or chest pain. 

Warnings & Precautions

Who should not use midostaurin?

Allergies to Ingredients. People who are allergic to any of the following should not take midostaurin.

  • Midostaurin
  • Rydapt
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in the specific midostaurin products they stock.

What should I know about midostaurin before using it?

Do not take midostaurin unless it has been prescribed to you by a health care provider. Take it as prescribed.

Do not share midostaurin with other people, even if they have the same condition as you. It may harm them.

Keep midostaurin out of the reach of children.

Do not open or crush the capsules. 

Your health care provider will do regular blood tests while you are taking midostaurin to make sure the medicine is safe for you. Keep all appointments with your health care provider. 

Midostaurin may make it difficult for you to get pregnant or get your partner pregnant. If you have concerns, talk to your health care provider.

If you are able to get a partner pregnant, you should use condoms while taking midostaurin and for four months after your last dose. 

What should I tell my health care provider before using midostaurin?

Tell your health care provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them know if midostaurin is right for you.

In particular, make sure that you discuss any of the following.

Current and Past Health Conditions. Tell your health care provider if you have any of the following.

  • Lung or breathing problems
  • Heart problems

Other Medicines and Supplements. Midostaurin may interact with other medicines and supplements. Before taking midostaurin, tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. See the Interactions section for more details.

Pregnancy. Midostaurin may harm an unborn baby. Your health care provider will give you a test to make sure you are not pregnant before you start taking midostaurin. If you can get pregnant, you should use birth control while taking midostaurin and for four months after your last dose. Tell your health care provider if you are pregnant or plan to become pregnant.

If you are pregnant while taking midostaurin or became pregnant by a partner taking midostaurin, call the drugmaker at 888-669-6682 or go to www.report.novartis.com to take part in a pregnancy registry that will monitor how your pregnancy goes.

Breastfeeding. It is not known if midostaurin passes into breast milk. You should not breastfeed while taking midostaurin and for four months after your last dose. Tell your health care provider if you are breastfeeding or plan to breastfeed.

Interactions

Does midostaurin interact with foods or drinks?

Midostaurin should be taken with food. 

Grapefruit interacts with midostaurin. Do not eat grapefruit or drink grapefruit juice while taking midostaurin. This is because grapefruit can interfere with your body's ability to break down midostaurin, which can lead to high levels of the medicine and an increased risk of side effects. 

It is unknown if drinking alcohol will affect midostaurin, but heavy drinking can slow your immune response and make it harder for your body to fight cancer if you take this medicine for cancer.

Does midostaurin interact with other medicines (drug interactions)?

Always tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using.

In particular, make sure that you discuss if you are using any of the following before taking midostaurin.

  • Itraconazole (Sporanox, Tolsura), ketoconazole, posaconazole (Noxafil), or voriconazole (Vfend), which are medicines used for fungal infections
  • Rifampin (Rifadin), which is a medicine commonly used to treat an infection called tuberculosis
  • Bupropion (Wellbutrin SR, Wellbutrin XL, Aplenzin, and others), which is a medicine used to treat depression or to help quit smoking
  • Nelfinavir (Viracept) or ritonavir (Norvir), which are medicines commonly used to treat HIV
  • Clarithromycin, which is a medicine used for bacterial infections
  • Phenytoin (Dilantin, Phenytek, and others), which is a medicine to control seizures
  • Carbamazepine (Carbatrol, Tegretol, and others), which is a medicine commonly used to treat seizures or nerve pain
  • St. John's wort, which is a supplement that may be used to treat depression
  • Any medicine that can cause QT prolongation, which is a potentially dangerous heart rhythm problem

Many other medicines may alter the blood levels of midostaurin, and vice versa. Tell your health care provider about all medicines that you take or have recently taken

This may not be a complete list of medicines that can interact with midostaurin. Always check with your health care provider.

Does interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose/Missed Dose

What should I do if I accidentally use too much midostaurin?

If you or someone else has used too much midostaurin, get medical help right away, call 911, or contact a Poison Control center at 800-222-1222.

What should I do if I miss a dose of midostaurin?

If you miss a dose, take your next dose at the regular time. Do not take double or extra doses.

If you vomit after taking your midostaurin dose, do not take another dose. Skip the dose and take your next dose at the usual time.