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WASHINGTON -- The FDA today approved the FLT3 inhibitor midostaurin (Rydapt) as a component of combination therapy for patients with a subtype of newly diagnosed acute myelogenous leukemia (AML).

The approval covers use of midostaurin in combination with chemotherapy for patients with FLT3-mutated AML. The approval, which follows the FDA's designation of midostaurin for priority review in November 2016, requires use of a companion diagnostic test, the LeukoStrat CDx FLT3 Mutation Assay, approved simultaneously with the drug.

"Rydapt is the first targeted therapy to treat patients with AML, in combination with chemotherapy," Richard Pazdur, MD, acting director of the FDA Office of Hematology and Oncology Products, said in a statement. "The ability to detect the gene mutation with a diagnostic test means doctors can identify specific patients who may benefit from this treatment."

Initially evaluated as treatment for the broad population of patients with AML, midostaurin failed to demonstrate efficacy. However, targeting the drug, in combination with chemotherapy, to the subgroup of patients with FLT3-mutated disease changed impressions about midostaurin.

In a randomized trial involving 717 patients with newly diagnosed FLT3-mutated AML, the addition of midostaurin to chemotherapy led to improved survival and event-free survival as compared with chemotherapy alone. The most common adverse events associated with midostaurin treatment were febrile neutropenia, nausea, mucositis, vomiting, headache, petechiae, musculoskeletal pain, epistaxis, device-related infection, hyperglycemia, and upper respiratory tract infections.

Separately, the FDA also approved midostaurin for a group of related rare blood disorders (aggressive systemic mastocytosis, systemic mastocytosis with associated hematologic neoplasm, and mast cell leukemia). Common adverse events associated with midostaurin treatment of these patients included nausea, vomiting, diarrhea, edema, musculoskeletal pain, abdominal pain, fatigue, upper respiratory infection, constipation, fever, headache, and dyspnea.

Novartis received approval for midostaurin and Invivoscribe Technologies for the companion diagnostic test.

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