The U.S. Food and Drug Administration (FDA) approved midostaurin in combination with chemotherapy for the treatment of adults with newly diagnosed FLT3-mutated acute myeloid leukemia (AML). Midostaurin is the first FDA-approved targeted therapy for AML.
The approval was based on results from the phase III RATIFY trial, in which 717 patients were randomized to receive chemotherapy (daunorubicin plus cytarabine) with or without midostaurin. Although a median survival rate could not yet be estimated, patients who received the combination therapy had longer event-free survival than those who received chemotherapy alone (median = 8.2 months and 3 months, respectively). Events were failure to achieve complete remission within 60 days of starting treatment, progression of leukemia, or death.
Common adverse events associated with midostaurin treatment included febrile neutropenia, nausea, mucositis, vomiting, headache, petechiae, musculoskeletal pain, nosebleed, device-related infection, hyperglycemia, and upper respiratory tract infection.
The FDA had previously granted midostaurin priority review, fast-track designation, and breakthrough-therapy designation.
Source: FDA news release, April 28, 2017.