Midostaurin
Midostaurin is an oral targeted therapy classified as a multikinase inhibitor. It is primarily used to treat acute myeloid leukemia (AML) in patients with FLT3 mutations and certain forms of systemic mastocytosis. The medication works by inhibiting specific enzymes that drive cancer cell growth and survival.
Midostaurin is a targeted therapy for FLT3-mutated AML and systemic mastocytosis that inhibits key enzymes to stop cancer cell growth.
Key facts at a glance
- ATC
- L01EX10
- RxNorm
- 1919083
- Generic
- Midostaurin
- Class
- atc-l01
- Brands
- Rydapt
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- capsule
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Midostaurin is approved for newly diagnosed FLT3-mutated acute myeloid leukemia (used with chemotherapy) and for treating advanced systemic mastocytosis, including aggressive systemic mastocytosis and mast cell leukemia.
Midostaurin inhibits multiple tyrosine kinases including FLT3 and KIT, blocking signaling pathways involved in cancer cell proliferation, survival, and differentiation. This targeted approach helps slow or stop the growth of cancer cells.
Midostaurin is taken orally, typically twice daily with food. Common side effects include nausea, vomiting, diarrhea, and low blood counts. It requires careful monitoring and should only be prescribed by specialists experienced in treating the indicated conditions. Patients should be monitored for signs of differentiation syndrome, a potentially serious complication.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed August 20, 2026. · Read our editorial policy