FDA approves Rydapt, first targeted AML drug

FDA approved Rydapt midostaurin from Novartis AG (NYSE:NVS; SIX:NOVN) to treat newly diagnosed acute myelogenous leukemia with an FMS-like tyrosine kinase 3 (FLT3; CD135) mutation. Rydapt is the first targeted AML therapy to gain FDA approval, and the first drug approved in the U.S. to treat AML in more than 40 years (see BioCentury, Dec. 9, 2016).

The agency approved Rydapt in combination with chemotherapy. FDA also approved the drug as monotherapy to treat advanced systemic mastocytosis, including aggressive systemic mastocytosis; systemic mastocytosis with associated hematological neoplasm; and mast cell leukemia. Rydapt is an FLT3 and stem cell factor (SCF) receptor tyrosine kinase (c-Kit; KIT; CD117) inhibitor. ...