FDA Approves Midostaurin to Treat Acute Myeloid Leukemia

Midostaurin demonstrated a statistically significant improvement in overall survival among AML patients.

Midostaurin (Rydapt) was approved by the FDA for the treatment of adults with newly diagnosed acute myeloid leukemia (AML), who are FLT3 mutation-positive in combination with cytarabine and daunorubicin induction and cytarabine consolidation.

The approval was based on a randomized, double-blind, placebo-controlled clinical trial that included 717 treatment-naive patients with FLT3+ AML, according to a press release.

Participants were randomized to receive either placebo or 50 mg of midostaurin orally twice-daily on days 8 to 21 of each cycle of induction and consolidation chemotherapy followed by continuous daily midostaurin for up to 12 cycles.

The results of the study showed a statistically significant improvement in overall survival for patients who received midostaurin compared with patients who received placebo.

Common adverse events (AEs) occurred in at least 20% of patients, and included febrile neutropenia, nausea, mucositis, vomiting, headache, petechiae, musculoskeletal pain, epistaxis, device-related infection, hyperglycemia, and upper respiratory infection.

Serious AEs were febrile neutropenia, which occurred in 16% of patients in both arms, according to the release.

Additionally, midostaurin was granted FDA approval for the treatment of adults with aggressive systemic mastocytosis (SM), SM with associated hematological neoplasm, or mast cell leukemia.

The approval was based on a single-arm, open-label study of 100 mg of midostaurin orally twice-daily. The results of the study showed that after 6 cycles of midostaurin, the rates of confirmed complete remission (CR) plus incomplete remission by modified Valent criteria were 38% for ASM and 16% for SM-AHN. There was 1 patient with mast cell leukemia who achieved a CR.

Common AEs included nausea, vomiting, diarrhea, edema, musculoskeletal pain, abdominal pain, fatigue, upper respiratory tract infection, fever, headache, and dyspnea.

The FDA also approved LeukoStrat CDx FLT3 Mutations Assay as a companion diagnostic for use with midostaurin to test patients with AML for the FLT3 mutation.