FDA grants Breakthrough status for Novartis' midostaurin in AML indication

Swiss pharma major Novartis (NOVN: VX) revealed on Friday that the US Food and Drug Administration has granted Breakthrough Therapy designation to its PKC412 (midostaurin).

Midostaurin is an investigational treatment for adults with newly-diagnosed acute myeloid leukemia (AML) who are FLT3 mutation-positive, as detected by an FDA-approved test, and who are eligible to receive standard induction and consolidation chemotherapy.

The Breakthrough Therapy designation for midostaurin is primarily based upon the positive results from the Phase III RATIFY (CALGB 10603) clinical trial. This study was conducted in partnership with the Alliance for Clinical Trials in Oncology and presented during a plenary session at the 57th American Society of Hematology (ASH) Annual Meeting.