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

Midostaurin 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.

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

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

Your doctor will perform a test to make sure midostaurin 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.

Midostaurin 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 midostaurin.

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

Do not crush or open a midostaurin capsule.

You may need frequent blood tests (every 1 to 4 weeks). Midostaurin 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 midostaurin in the original container at room temperature, away from moisture and heat.

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 cytarabine consolidation
chemotherapy, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (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

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.
-Treatment should be continued until disease progression or unacceptable toxicity.

Uses: For the treatment of adult patients with 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

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.
-Treatment should be continued until disease progression or unacceptable toxicity.

Uses: For the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic
mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL)

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

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 if the mother or the father is using this medicine.

• 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 midostaurin.

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 midostaurin can harm an unborn baby.

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

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

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.

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

Keep this medication in the container it came in, tightly closed, and out of reach of children. Store it at room temperature and away from excess heat and moisture (not in the bathroom).

Unneeded medications should be disposed of in special ways to ensure that pets, children, and other people cannot consume them. However, you should not flush this medication down the toilet. Instead, the best way to dispose of your medication is through a medicine take-back program. Talk to your pharmacist or contact your local garbage/recycling department to learn about take-back programs in your community. See the FDA's Safe Disposal of Medicines website (http://goo.gl/c4Rm4p) for more information if you do not have access to a take-back program.

Talk to your doctor about eating grapefruit and drinking grapefruit juice while taking this medication.

Unless your doctor tells you otherwise, continue your normal diet.

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

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

Midostaurin may cause serious side effects. 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 side effects of midostaurin 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.

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 FDA at 1-800-FDA-1088.

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 affect midostaurin, 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.

Keep all appointments with your doctor and the laboratory. Your doctor may order certain lab tests to check your body's response to midostaurin.

It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.