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# Novartis Gains Priority Review for Midostaurin in Leukemia
- URL: https://www.fdaweb.com/novartis-gains-priority-review-for-midostaurin-in-leukemia/
- Published: 2016-11-14T12:00:00.000Z
- Updated: 2026-09-14T21:47:17.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5137336

FDA has granted Novartis a priority review for an NDA for midostaurin, indicated for treating acute myeloid leukemia (AML) in newly-diagnosed adults with an FMS-like tyrosine kinase-3 (FLT3*)* mutation, as well as for treating advanced systemic mastocytosis (SM). A PMA for the midostaurin FLT3 companion diagnostic, developed in collaboration with Invivoscribe Technologies, has also been accepted for review by the agency.

The NDA includes data from the Phase III RATIFY trial (CALGB 10603), which investigated midostaurin plus standard chemotherapy versus placebo plus standard chemotherapy in adult patients less than 60 years of age with *FLT3*\-mutated AML. Those in the midostaurin arm experienced a statistically significant improvement in overall survival with a 23% reduction in risk of death compared to placebo, Novartis says. It also includes data from a Phase 2 single-arm study evaluating midostaurin’s efficacy in patients with advanced SM. The study showed that treatment with midostaurin resulted in an overall response rate of 60% (defined as complete or partial resolution of organ damage) with a median duration of response of 24.1 months, it says.