Novartis’ precision medicine for acute myeloid leukaemia (AML) has been granted breakthrough therapy designation by the FDA.

PKC412 (midostaurin) is specifically designed to treat AML patients with a FLT3 gene mutation. Approximately one third of AML patients harbour this mutation, which is associated with worse outcomes and shorter survival than in those without the mutation.

The breakthrough therapy designation is based upon positive results from the Phase III RATIFY clinical trial. Patients who received PKC412 and standard induction and consolidation chemotherapy experienced a significant improvement in overall survival compared to those who received standard induction and consolidation chemotherapy alone. The median overall survival for patients in the PKC412 treatment group was 74.7 months, versus 25.6 months in the placebo group. 

"For more than 25 years, medical developments have been limited for AML patients and the chemotherapy treatment strategy has essentially remained unchanged," said Alessandro Riva, MD, Global Head, Novartis Oncology Development and Medical Affairs. "We look forward to working closely with the FDA to bring the first potential AML targeted therapy to patients as quickly as possible."

Novartis is also collaborating with Invivoscribe Technologies, who are leading regulatory submissions for a companion diagnostic that could help identify patients who may have a FLT3 mutation.