Rydapt Approved for Acute Myeloid Leukemia and Systemic Mastocytosis
April 28, 2017 – The U.S. FDA approved for Rydapt® (midostaurin), manufactured by Novartis. Rydapt is indicated for the first-line treatment of adults with FMS-like tyrosine kinase 3 mutation-positive (FLT3+) acute myeloid leukemia (AML) as detected by an FDA-approved test, in combination with chemotherapy. It is also approved to treat adults with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN) or mast cell leukemia (MCL). The recommended dose for AML is 50mg twice daily with food. For ASM, SM-AHN and MCL, the recommended dose is 100mg twice daily with food. The estimated cost is $180,000 per year (wholesale acquisition cost).
AML is a rare and aggressive cancer of the blood and bone marrow. Approximately 21,000 patients are diagnosed with AML each year in the United States. About one-third of these patients have a FLT3 gene mutation, which is associated with lower survival rates than other forms of AML. Rydapt is the only targeted therapy approved to treat patients with newly diagnosed FLT3+ AML. In a Phase III clinical trial of newly diagnosed patients with FLT3+ AML, Rydapt in combination with chemotherapy improved overall survival by 23% compared to chemotherapy alone.
Advanced systemic mastocytosis is a rare blood disorder that is associated with the uncontrolled growth and accumulation of mast cells in one or more organs. This can lead to organ failure and death within six months to three and a half years, depending on subtype. Approval for advanced mastocytosis was based on a Phase II study that demonstrated an overall response rate of 21%.
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