Novartis drug PKC412 gets Breakthrough designation for AML
pharmafile | February 19, 2016 | News story | Medical Communications, Research and Development | FDA, Novartis, acute myeloid leukaemia
Novartis’ PKC412 (midostaurin) has become the latest in a sequence of FDA Breakthrough Therapy designations awarded this week- bagging the status for its drug PKC412, to treat newly-diagnosed FLT3-mutated acute myeloid leukaemia (AML).
The investigational treatment would be given to adults with newly-diagnosed AML who test positive for the FLT3 mutation after an FDA-approved test, and who are eligible to receive standard induction and consolidation chemotherapy. Research suggests that about of third of AML patients harbour the FLT3 mutation, which is associated with worse outcomes and shorter survival than those without.
Novartis says it is collaborating with Invivoscribe Technologies, which is leading the race to regulatory approval for a companion diagnostic test to help identify such patients, who may benefit most from PKC412 in treating the aggressive blood cancer.
The Breakthrough Therapy designation is based largely on the positive results from a Phase III trial, in which 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. Mean overall survival for the PKC412-treated patients was 74.7 months, compared to an average of 25.6 months for patients in the placebo group.
Alessandro Riva, global head, Novartis Oncology Development and Medical Affairs, welcomed the decision as bringing a long-awaited new treatment option a step closer to patients. He comments: “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.”
Some 20,000 people were diagnosed with AML in the US in 2015. The condition, affecting the blood and bone marrow, causes an accumulation of ‘blasts’ which do not allow room for the normal blood cells. AML is the most common acute leukaemia in adults, but also has the lowest survival rate.
Joel Levy
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