The FDA has granted priority review for Novartis’ new drug application (NDA) for their orphan drug PKC412.
PKC412 is intended for the treatment of acute myeloid leukemia (AML) in newly-diagnosed adults with an FMS-like tyrosine kinase-3 (FLT3) mutation, as well as for the treatment of advanced systemic mastocytosis (SM).
The submission of the NDA includes data from Phase 2 and 3 trials of PKC412. In the Phase 3 trial, PKC412 (midostaurin) was investigated with standard chemotherapy versus placebo plus standard chemotherapy in adult patients less than 60 years of age with FLT3-mutated AML.
Patients in the PKC412 arm experienced a statistically significant improvement in overall survival (OS) with a 23% reduction in risk of death compared to the placebo arm (hazard ratio [HR] = 0.77, P = 0.0074).
Because of this encouraging data, PKC412 was also granted Breakthrough Therapy designation by the FDA earlier this year.
PKC412 (midostaurin) is an oral, multi-targeted kinase inhibitor (targeting both wild type KIT and the KIT D816V mutation, among others) for the treatment of AML with a FLT3 mutation, as well as patients with advanced SM. If approved, this will be the first drug to treat FLT3 mutated AML.
About Acute Myeloid Leukemia
acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes abnormal myeloblasts (a type of white blood cell), red blood cells, or platelets.
AML can sometimes spread to other parts of the body including the lymph nodes, liver, spleen, central nervous system (brain and spinal cord), and testicles. Symptoms include fatigue, recurrent infections, and bruising easily.