Novartis’ PKC412 granted Breakthrough status for AML

Novartis’ acute myeloid leukaemia (AML) drug PKC412 has gained Breakthrough status following the release of phase 3 trial results.

The multi-kinase protein inhibitor PKC412 (midostaurin) is specifically designed to combat AML in patients with a FLT3 genetic mutation.

The US FDA’s Breakthrough Therapy Designation followed the presentation of promising results from the phase 3 RATIFY trial, which demonstrated an increase of median overall survival of almost 50 months.

The trial investigated the use of PKC412 plus standard induction and consolidation chemotherapy versus standard chemotherapy alone.

The PKC412 group demonstrated an average overall median survival of 74.7 months, in stark contrast to the 25.6 month average seen in the standard treatment group.

Alessandro Riva, MD, Global Head, 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 (midostaurin), the first potential AML targeted therapy, to patients as quickly as possible.”

This latest Breakthrough designation continues the trend seen in Novartis’ relatively successful 2015, which started with the unanimous approval of its Neupogen biosimilar Zarxio.

In 2015, AML affected around 20,000 people in the US alone, of which, an estimated one third harboured an FLT3 gene mutation. The mutation has been linked with worse outcomes and shorter survival than those without it. PKC412 is now the first FLT3 targeted therapy to demonstrate an improvement in overall AML survival.

Novartis is currently working with Invivoscribe Technologies on the development of a companion diagnostic that may help identify patients with an FLT3 mutation.

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