mstarin midostaurin

Rydapt (Midostaurin): FDA Approval, Uses, and What You Need to Know

Rydapt, whose active ingredient is midostaurin, is a targeted oncology drug developed by Novartis. It has recently gained FDA approval for two major indications: treatment of newly diagnosed FLT3-mutated acute myeloid leukemia (AML) in combination with chemotherapy, and several types of advanced systemic mastocytosis (SM). 

What Is FLT3-Mutated AML & Why Rydapt Matters

• Acute myeloid leukemia (AML) is a fast-progressing blood cancer where immature white blood cells accumulate and impair normal blood formation. Roughly 25-30% of AML patients have a mutation in the FLT3 gene, which is associated with worse outcomes. 

• Until Rydapt’s approval, standard AML treatment hadn’t changed significantly in over 25 years for patients with FLT3 mutations. Rydapt, in combination with standard chemotherapy (cytarabine + daunorubicin induction and cytarabine consolidation), showed a 23% reduction in risk of death in the RATIFY trial compared to chemotherapy alone. 

Advanced Systemic Mastocytosis (SM)

• SM is a rare disease characterized by abnormal proliferation of mast cells, leading to organ damage. “Advanced SM” includes aggressive systemic mastocytosis (ASM), SM with associated hematologic neoplasm (SM-AHN), and mast cell leukemia. 

• Rydapt is now the first approved therapy for these types of SM, offering a treatment where options are very limited.

Dosage & Usage: How Rydapt Is Used

According to prescribing information:

• For newly diagnosed FLT3-mutated AML, Rydapt is given orally at 50 mg twice daily, starting on day 8 through day 21 of each induction and consolidation chemotherapy cycle. 

• For advanced SM, the dose is 100 mg twice daily. 

Patients should follow an FDA-approved test for FLT3 mutation before using Rydapt in AML. It is not recommended as a single-agent induction therapy in AML. 

Side Effects & Safety Information

Common adverse reactions when Rydapt is used in AML + chemotherapy include:

• Febrile neutropenia (low white blood cells + fever)

• Nausea, vomiting, mucositis (mouth sores)

• Headache, musculoskeletal pain, hyperglycemia

• Device-related infections 

For SM, frequent side effects include diarrhea, swelling, fatigue, upper respiratory infection, etc. Serious risks include allergic reactions, lung problems, effects on liver (‘hepatotoxicity’), and effects on fertility. Pregnancy is contraindicated; both men and women should use effective contraception during treatment and for several months after. 

Why This FDA Approval Is a Milestone

• It is the first new treatment in over 25 years for FLT3-mutated AML. 

• Rydapt significantly improves overall survival and event-free survival compared to chemotherapy alone, setting a new standard of care. 

• For advanced SM patients, who previously had no approved targeted therapies, Rydapt now provides a clinically validated option.

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