FDA Grants Priority Review to Midostaurin for Newly Diagnosed AML

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The FDA has granted Priority Review to the NDA for midostaurin (PKC412) for the treatment of adults with acute myeloid leukemia.
The FDA has granted Priority Review to the NDA for midostaurin (PKC412) for the treatment of adults with acute myeloid leukemia.

The U.S. Food and Drug Administration (FDA) has granted Priority Review to the New Drug Application (NDA) for midostaurin (PKC412) for the treatment of adults with newly diagnosed FML-like tyrosine kinase-3 (FLT3)-mutated acute myeloid leukemia (AML), as well as for the treatment of advanced systemic mastocytosis, according to an announcement by Novartis.1

The FDA has also accepted the premarket approval (PMA) application for a midostaurin FLT3 companion diagnostic test, developed in collaboration with the Invivoscribe Technologies, Inc.

Submission of this NDA is based on findings from the phase 3 RATIFY trial (CALGB 10603), which showed that patients aged younger than 60 years with newly diagnosed FLT3-mutated AML who received midostaurin plus chemotherapy had a 23% reduced risk of death compared with those who received chemotherapy alone (hazard ratio, 0.77; P = .0074).

There were no significant differences in overall rate of grade 3 or higher hematologic and non-hematologic adverse events between the midostaurin plus chemotherapy arm and the chemotherapy only arm. The most common adverse events in midostaurin group were febrile neutropenia, nausea, exfoliative dermatitis, vomiting, headache, petechiae, and pyrexia.

RELATED: "Beat AML" Master Trial Aims To Be Leader in Precision Medicine

The NDA also includes data from a single-arm, phase 2 trial evaluating the efficacy and safety of midostaurin in patients with advanced systemic mastocytosis. The study demonstrated an overall response rate of 60% and a median overall survival of 28.7 months (95% CI, 18.1-not evaluable).

The FDA is expected to take action on this NDA within 6 months, as opposed to 10 months under the standard review process. 

Reference

  1. The U.S. Food and Drug Administration (FDA) has granted Priority Review to the New Drug Application (NDA) for midostaurin (PKC412) for the treatment of adults with newly diagnosed FML-like tyrosine kinase-3 (FLT3)-mutated acute myeloid leukemia (AML), as well as for the treatment of advanced systemic mastocytosis, according to an announcement by Novartis.1

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