Experimental Novartis leukaemia and mastocytosis drug gets FDA Priority Review

pharmafile | November 14, 2016 | News story | Research and Development, Sales and Marketing FDA, Novartis, acute myeloid leukaemia 

The US FDA has granted Priority Review status to Novartis’ experimental drug PKC412 (midostaurin) as a treatment for advanced systemic mastocytosis (SM) in addition to acute myeloid leukaemia (AML) in adult patients with an FMS-like tyrosine kinase-3 (FLT3) mutation.

The decision hinged upon Phase III data generated from the largest clinical trials ever conducted in each indication, which showed that patients treated with the drug showed improved overall survival rates with a risk of death reduction of 23% compared to placebo. No significant differences in the rate of grade 3+ hematologic and non-hematologic adverse events were recorded. This same data also won the drug its Breakthrough Therapy designation by the FDA earlier this year.

CEO of Novartis Oncology Bruno Strigini commented: “FLT3-mutated AML and advanced SM are devastating and rare diseases, with significant unmet needs due to limited existing treatment options. This regulatory designation signifies the importance of midostaurin as a potential therapy for these patients who haven’t had the benefit of targeted medicines.”

The drug has already been accepted for the same indications by the European Medicines Agency (EMA). Novartis has opened both a Global Individual Patient Programme and an Expanded Treatment Protocol to ensure eligible patients have access to the drug.

Matt Fellows

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