The US Food and Drug Administration (FDA) has granted breakthrough therapy designation to PKC412 (midostaurin), 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.

In the US, about 20,000 people were diagnosed with AML in 2015, the majority of whom were adults. According to research, approximately one-third of AML patients also harbor a FLT3 gene mutation, which is associated with worse outcomes and shorter survival than in those without the mutation. PKC412 (midostaurin) is a drug targeting FLT3 to demonstrate an overall survival benefit in AML.

PKC412 (midostaurin) is also being investigated for the treatment of aggressive systemic mastocytosis/mast cell leukemia.

Source: Novartis