The FDA grants midostaurin (Rydapt) approval for the treatment of newly diagnosed FLT3+ AML patients

On 28^th April 2017, the U.S. Food and Drug Administration (FDA) granted approval to midostaurin (Rydapt) for the treatment of newly diagnosed Acute Myeloid Leukemia (AML) who are Fms Like Tyrosine Kinase 3 (FLT3) mutated-positive (FLT3+), as detected by an FDA-approved test, in combination with standard cytarabine and daunorubicin and cytarabine consolidation.¹

In addition, the FDA approved a companion diagnostic, the LeukoStrat CDx FLT3 Mutation Assay for use with midostaurin to test patients with AML for the FLT3 mutations.

This approval for midostaurin, a FLT3 inhibitor, is based on results from the phase 3 RATIFY trial (NCT00651261), which as a randomized trial in 717 newly diagnosed FLT3+ AML patients. In this study, patients were randomly assigned to receive either placebo or midostaurin 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.

The key results of the study were:

• Overall Survival (OS) in patients receiving midostaurin and placebo; 74.4 vs 25.6 months, HR = 77, P = 0.0074
• Adverse Events (AEs) occurred in at least 20% of patients, included febrile neutropenia, nausea, mucositis, vomiting, headache, petechiae, musculoskeletal pain, epistaxis, device-related infection, hyperglycemia, and upper respiratory tract infection.

The approved regimens of midostaurin in AML is 50 mg twice daily with food on days 8 to 21 of each cycle of induction and consolidation chemotherapy followed by 50 mg with food as a single agent for up to 12 months.