The US Food and Drug Administration (FDA) approved midostaurin as a treatment for patients with newly diagnosed, FLT3 mutation-positive acute myeloid leukemia (AML), according to a press release.1

A companion diagnostic test — the LeukoStrat CDx FLT3 Mutation Assay — was also approved to detect FLT3 mutations among patients with AML.

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This approval was based on the results of a phase 3 trial (Daunorubicin, Cytarabine, and Midostaurin in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia; Identifier: NCT00651261) showing a significant overall survival benefit for patients randomly assigned to receive midostaurin over those assigned to placebo.

The hazard ratio for overall survival between the midostaurin group vs placebo was 0.77 (P = .016). The study included 717 patients with newly diagnosed, FLT3-positive AML.

The press release notes common adverse events observed with midostaurin, which include “febrile neutropenia, nausea, mucositis, vomiting, headache, petechiae, musculoskeletal pain, epistaxis, device-related infection, hyperglycemia, and upper respiratory tract infection.”

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Midostaurin must be used with cytarabine and daunorubicin induction as well as cytarabine consolidation.

Prescribing information can be found here.


  1. Midostaurin [news release]. Silver Spring, MD: US Food and Drug Administration; April 28, 2017. Accessed April 28, 2017.