Novartis' acute myeloid leukaemia (AML) treatment PKC412 (midostaurin) has been granted 'breakthrough therapy' status by the FDA.
PKC412 is as an investigational oral kinase inhibitor for the treatment of adults with newly-diagnosed AML and the FTL3 mutation, eligible to undergo standard induction and consolidation chemotherapy.
The US regulator reached the decision after PKC412 was shown to significantly improve overall survival of adult patients to who received chemotherapy treatment for AML in a phase III trial.
First presented at the American Society of Hematology (ASH) conference last year, the phase III RATIFY clinical trial found that PKC412 extended post-treatment survival to 74.7 months in comparison with 25.6 months for patients in the placebo group.
An aggressive cancer of the blood and bone marrow, AML is the most common acute leukaemia in adults accounting for approximately 25% of adult leukaemia worldwide, but also the most fatal.
One third of AML patients also harbour the FLT3 mutation - a receptor tyrosine kinase that increases the number of certain blood cells - and is associated with a lower survival rate still.
PKC412 is the first AML drug to also target the FLT3 mutation that has demonstrated an increase in survival expectancy though its safety and efficacy profile has not yet been fully established.
Alessandro Riva, managing director and global head for Novartis' oncology development and medical affairs, said: “For more than 25 years, medical developments have been limited for AML patients and the chemotherapy treatment strategy has essentially remained unchanged.
“We look forward to working closely with the FDA to bring PKC412 … to patients as quickly as possible.”
The breakthrough designation will expedite the development and review of PKC412 by the FDA as well as provide Novartis with a higher level of guidance on efficient drug development.
PKC412 is currently also being investigated for the treatment of aggressive systemic mastocytosis/mast cell leukaemia.
Should it reach the market PKC412 will compete with the likes of Celgene's Vidaza (azacitidine), which was first approved in 2008 and last November won an expanded indication to treat elderly patients with AML.
Also waiting in the wings could be volasertib from Boehringer Ingelheim, which is currently in phase III clinical trials, and Pifzer's Mylotarg, which would be reintroduced to the market after positive phase III results.
Meanwhile, Novartis says its PKC412 is on track for global regulatory submissions later this year.