Pronunciation:

mye-doe-STAW-rin

Brand Names:

• Rydapt

Dosage Forms:

• Capsule, Liquid Filled

Classifications:

Therapeutic—

Antineoplastic Agent

Uses of This Medicine:

Midostaurin is used in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation chemotherapy to treat newly diagnosed acute myeloid leukemia (AML) in patients who are FLT3 mutation-positive. Your doctor will perform a test to check for this mutation before you use this medicine.

This medicine is also used to treat aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL).

This medicine is available only with your doctor's prescription.

Before Using This Medicine:

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Children—

Appropriate studies have not been performed on the relationship of age to the effects of midostaurin in the pediatric population. Safety and efficacy have not been established.

Older adults—

Appropriate studies performed to date have not demonstrated geriatric-specific problems that would limit the usefulness of midostaurin in the elderly. However, elderly patients are more likely to have age-related medical problems, which may require caution and an adjustment in the dose for patients receiving this medicine.

Breast-feeding—

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Other medicines—

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. When you are taking this medicine, it is especially important that your healthcare professional know if you are taking any of the medicines listed below. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.

Using this medicine with any of the following medicines is not recommended. Your doctor may decide not to treat you with this medication or change some of the other medicines you take.

• Bacillus of Calmette and Guerin Vaccine, Live
• Dengue Tetravalent Vaccine, Live
• Measles Virus Vaccine, Live
• Mumps Virus Vaccine, Live
• Rubella Virus Vaccine, Live
• Smallpox Monkeypox Vaccine
• Typhoid Vaccine
• Varicella Virus Vaccine
• Yellow Fever Vaccine

Using this medicine with any of the following medicines is usually not recommended, but may be required in some cases. If both medicines are prescribed together, your doctor may change the dose or how often you use one or both of the medicines.

• Adagrasib
• Adenovirus Vaccine Type 4, Live
• Adenovirus Vaccine Type 7, Live
• Apalutamide
• Asciminib
• Atorvastatin
• Avacopan
• Belzutifan
• Berotralstat
• Boceprevir
• Bosentan
• Bupropion
• Carbamazepine
• Ceritinib
• Cholera Vaccine
• Clarithromycin
• Cobicistat
• Conivaptan
• Crizotinib
• Dabrafenib
• Daridorexant
• Ebola Zaire Vaccine, Live
• Elagolix
• Enasidenib
• Encorafenib
• Enzalutamide
• Fedratinib
• Fluconazole
• Fosphenytoin
• Givinostat
• Glyburide
• Grazoprevir
• Idelalisib
• Indinavir
• Influenza Virus Vaccine, Live
• Irinotecan
• Itraconazole
• Ketoconazole
• Lanreotide
• Lazertinib
• Lefamulin
• Letermovir
• Lonafarnib
• Lopinavir
• Lumacaftor
• Mavorixafor
• Methotrexate
• Mitapivat
• Mitotane
• Mobocertinib
• Nefazodone
• Nelfinavir
• Nirogacestat
• Octreotide
• Ozanimod
• Pacritinib
• Palbociclib
• Phenytoin
• Pitavastatin
• Posaconazole
• Pravastatin
• Repaglinide
• Repotrectinib
• Revefenacin
• Ribociclib
• Rifampin
• Ritlecitinib
• Ritonavir
• Rosuvastatin
• Rotavirus Vaccine, Live
• Saquinavir
• Selpercatinib
• Simvastatin
• Sotorasib
• St John's Wort
• Sulfasalazine
• Suzetrigine
• Taurursodiol
• Tazemetostat
• Telaprevir
• Telithromycin
• Tenofovir Alafenamide
• Tocilizumab
• Topotecan
• Tovorafenib
• Treosulfan
• Tucatinib
• Ubrogepant
• Vonoprazan
• Vorasidenib
• Voriconazole
• Voxelotor

Using this medicine with any of the following medicines may cause an increased risk of certain side effects, but using both drugs may be the best treatment for you. If both medicines are prescribed together, your doctor may change the dose or how often you use one or both of the medicines.

• Mavacamten

Other interactions—

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.

Using this medicine with any of the following is usually not recommended, but may be unavoidable in some cases. If used together, your doctor may change the dose or how often you use this medicine, or give you special instructions about the use of food, alcohol, or tobacco.

• Grapefruit Juice

Other medical problems—

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

• Lung disease (eg, interstitial lung disease, pneumonitis)—Use with caution. May make these conditions worse.

Proper Use of This Medicine:

Medicines used to treat cancer are very strong and can have many side effects. Before receiving this medicine, make sure you understand all the risks and benefits. It is important for you to work closely with your doctor during your treatment.

Take this medicine only as directed by your doctor. Do not use more of it, do not use it more often, and do not use it for a longer time than your doctor ordered.

This medicine should come with a patient information leaflet. Read and follow the instructions carefully. Ask your doctor if you have any questions.

Swallow the capsule whole. Do not crush, break, chew, or open it. Take this medicine with food.

You may also receive medicines to help prevent nausea and vomiting.

Do not eat grapefruit or drink grapefruit juice while you are using this medicine.

Dosing—

The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.

The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.

• For oral dosage form (capsules):
  • For treatment of acute myeloid leukemia:
    • Adults—50 milligrams (mg) 2 times a day on Days 8 to 21 of each cycle of induction with cytarabine and daunorubicin, and on Days 8 to 21 of each cycle of consolidation with high-dose cytarabine.
    • Children—Use and dose must be determined by your doctor.
  • For treatment of ASM, SM-AHN, and MCL:
    • Adults—100 milligrams (mg) 2 times a day.
    • Children—Use and dose must be determined by your doctor.

Missed dose—

If you miss a dose of this medicine, skip the missed dose and go back to your regular dosing schedule. Do not double doses.

If you vomit after taking your medicine, skip the missed dose and go back to your regular dosing schedule.

Storage—

Keep out of the reach of children.

Do not keep outdated medicine or medicine no longer needed.

Ask your healthcare professional how you should dispose of any medicine you do not use.

Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light. Keep from freezing.

Store the medicine in the original package to protect from moisture.

Precautions While Using This Medicine:

It is very important that your doctor check your progress at regular visits to make sure this medicine is working properly and to check for unwanted effects. Blood tests may be needed to check for unwanted effects.

Using this medicine while you are pregnant can harm your unborn baby. It may also cause birth defects if the father is using it when his sexual partner becomes pregnant. If you are a woman who can get pregnant, your doctor may do tests to make sure you are not pregnant within 7 days before starting treatment. Use an effective form of birth control to keep from getting pregnant during treatment and for 4 months after your last dose. Males who are taking this medicine, with female partners who can become pregnant must use effective birth control during treatment and for 4 months after the last dose of this medicine. If you think you have become pregnant while using the medicine, tell your doctor right away.

Tell your doctor if you have chest pain, chills, cough, fever, general feeling of discomfort or illness, thickening of bronchial secretions, or trouble breathing. These may be symptoms of a serious lung problem.

This medicine lowers the number of some types of blood cells in your body. Because of this, you may bleed or get infections more easily. To help with these problems, avoid being near people who are sick or have infections. Wash your hands often. Stay away from rough sports or other situations where you could be bruised, cut, or injured. Brush and floss your teeth gently. Be careful when using sharp objects, including razors and fingernail clippers.

Talk with your doctor before using this medicine if you plan to have children. Some men and women who use this medicine have become infertile (unable to have children).

Cancer medicines can cause nausea or vomiting in most people, sometimes even after receiving medicines to prevent it. Ask your doctor or nurse about other ways to control these side effects.

Do not take other medicines unless they have been discussed with your doctor. This includes prescription or nonprescription (over-the-counter [OTC]) medicines and herbal (eg, St. John's wort) or vitamin supplements.

Side Effects of This Medicine:

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur:

More common

Black, tarry stools

bloody urine

body aches or pain

bone, joint, or muscle pain

chest pain or tightness

cough

decreased frequency or amount of urine

dizziness

ear congestion

fever or chills

headache

increased thirst

loss of appetite

loss of voice

lower back or side pain

nausea

painful or difficult urination

pale skin

runny or stuffy nose

sneezing

sore throat

stomach pain

swelling of the face, fingers, or lower legs

trouble breathing

ulcers, sores, or white spots in the mouth

unusual bleeding or bruising

unusual tiredness or weakness

vomiting

weight gain

Incidence not known

Chills

general feeling of discomfort or illness

thickening of bronchial secretions

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

Bloody nose

blurred vision

cracked lips

diarrhea

difficulty having a bowel movement (stool)

difficulty with moving

difficulty with swallowing

dry mouth

flushed, dry skin

fruit-like breath odor

headache

increased hunger

increased sweating

increased thirst

increased urination

muscle stiffness

small red or purple spots on the skin

sores, ulcers, or white spots on the lips, tongue, or inside the mouth

sweating

trouble sleeping

unexplained weight loss

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.