The EMA grants midostaurin approval for the treatment of newly diagnosed FLT3+ AML patients

On 20^th September 2017, the European Commission (EC) granted approval to midostaurin in combination with standard daunorubicin and cytarabine induction and high-dose cytarabine consolidation chemotherapy, and for patients in complete response followed by Rydapt® single agent maintenance therapy, for adults with newly diagnosed Acute Myeloid Leukemia (AML) who are Fms Like Tyrosine Kinase 3 (FLT3) mutation-positive (FLT3+).¹

This approval comes after the ‘positive opinion’ recommendation of Rydapt® by the  European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) in July 2017 and the U.S. Food and Drug Administration (FDA) approval in April 2017.

The EC approval for midostaurin, a FLT3 inhibitor, was based on results from the phase III RATIFY trial (NCT00651261), which was a randomized trial in 717 newly diagnosed FLT3+ AML patients (median age = 47.9 years, range 18–59 years). In this study, patients were randomly assigned to receive either placebo or Rydapt® 50 mg orally twice daily on days 8–21 of each cycle of induction and consolidation chemotherapy followed by continuous daily midostaurin for up to 12 cycles. Median Overall Survival (OS) in patients receiving midostaurin and placebo were 74.7 and 25.6 months, respectively (HR for death = 0.78, P = 0.009).² More results of this study were reported here.

The drug manufacturers, Novartis, highlighted that midostaurin “represents the first and only targeted therapy for FLT3-mutated AML” in the European Union (EU).