Class: Biological Therapy

Generic Name: midostaurin

Trade Name: Rydapt®

For which conditions is this drug approved? Rydapt is approved for patients newly diagnosed with a certain type of acute myeloid leukemia (AML) in combination with certain chemotherapy medications, and for patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL).

What is the mechanism of action?  Patients with AML have a certain genetic mutation called FLT3. FLT3 is a receptor tyrosine kinase, a type of cell-surface receptor, which plays a role in the proliferation, or increase, in the number of certain blood cells. Rydapt is a kinase inhibitor that works by blocking several enzymes that promote cell growth.

How is Rydapt typically given? Rydapt is taken orally.

How are patients monitored? Patients will usually have scheduled meetings with their healthcare provider while they are being treated with Rydapt. Typically, blood will be drawn before and after treatment to check levels of blood cells and to monitor functions of some organ systems, such as the kidneys or liver.

What are the most common side effects of treatment with Rydapt?

For patients with AML:

• Low white cell blood counts with fever
• Nausea
• Redness, pain, or ulcers inside mouth
• Vomiting
• Headache
• Bruising
• Muscle or bone pain
• Nose bleeds
• Device-related infection
• High blood sugar levels
• Upper respiratory tract infection

For patients with ASM, SH-AHM, or MCL:

• Nausea
• Vomiting
• Diarrhea
• Swelling in hands, feet or ankles
• Muscle or bone pain
• Stomach-area pain
• Tiredness
• Upper respiratory tract infection
• Constipation
• Fever
• Headache
• Trouble breathing

What are some of the less common but potentially serious side effects of Rydapt?

• Lung problems.

This is not a complete list of side effects. Some patients may experience other side effects that are not listed here. Patients may wish to discuss with their physician the other less common side effects of this drug, some of which may be serious.

Some side effects may require medical attention. Other side effects do not require medical attention and may go away during treatment. Patients should check with their physician about any side effects that continue or are bothersome.

What should you tell your healthcare provider before starting treatment with Rydapt?

Tell your healthcare provider if you:

• Have any lung or breathing problems.
• Are pregnant or plan to become pregnant. Rydapt can harm your unborn baby or cause miscarriage.
• Are breastfeeding or plan to breastfeed.

Tell your health care provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Rydapt and other medicines may affect each other causing side effects. Know the medicines you take. Keep a list of them to show your health care provider or pharmacist when you get a new medicine.

When should patients notify their physician?

Contact your healthcare professional immediately in case of any of the following:

• You have symptoms of lung problems. These include: cough, chest discomfort, or shortness of breath.
• You become pregnant.

What is a package insert?

A package insert is required by the FDA and contains a summary of the essential scientific information needed for the safe and effective use of the drug for healthcare providers and consumers. A package insert typically includes information regarding specific indications, administration schedules, dosing, side effects, contraindications, results from some clinical trials, chemical structure, pharmacokinetics and metabolism of the specific drug. By carefully reviewing the package insert, you will get the most complete and current information about how to safely use this drug. If you do not have the package insert for the drug you are using, your pharmacist or physician may be able to provide you with a copy.

Last updated on 05/17.

Important Limitations of Use

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