publication date: Apr. 28, 2017

Drugs and Targets

FDA approves midostaurin combination for AML; Companion diagnostic also approved

FDA approved midostaurin, trade name Rydapt, for the treatment of adult patients with newly diagnosed acute myeloid leukemia who are FLT3 mutation-positive, as detected by an FDA-approved test, in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation.

The drug is sponsored by Novartis Pharmaceuticals Corp.

FDA also approved a companion diagnostic, the LeukoStrat CDx FLT3 Mutation Assay for use with midostaurin to test patients with AML for the FLT3 mutation. The diagnostic is sponsored by Invivoscribe Technologies Inc.

Approval was based on a randomized, double-blind, placebo-controlled trial in 717 patients with previously untreated FLT3+ AML. The trial randomized patients to 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 trial demonstrated a statistically significant improvement in overall survival (OS) for patients receiving midostaurin compared with those on the placebo-containing arm (HR 0.77, p=0.016).

Common adverse reactions, occurring 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 most frequent serious adverse reaction was febrile neutropenia, occurring in 16% of patients on both arms.

FDA also approved midostaurin for the treatment of adults with aggressive systemic mastocytosis, SM with associated hematological neoplasm, or mast cell leukemia.  Approval was based on response rate and duration in a single-arm, open-label study of midostaurin 100 mg orally twice daily. With 6 cycles of midostaurin, the rates of confirmed complete remission plus incomplete remission by modified Valent criteria were … Continue reading FDA approves midostaurin combination for AML; Companion diagnostic also approved

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