Novartis drug gets fast US review in AML

The FDA is to quickly review Novartis’s PKC412 (midostaurin) in the blood and bone marrow, acute myeloid leukaemia, with a certain mutation after the drug showed a survival benefit in a late-stage trial.

The US regulator will also review the drug in advanced systemic mastocytosis, a rare disease where growth of mast cells causing allergic responses is uncontrolled, leading to organ damage and other debilitating symptoms.

Novartis noted that the treatment strategy for AML has remained unchanged for more than 25 years, adding that midostaurin may represent the first drug to improve survival in FLT3-mutated disease.

The FDA has therefore granted a Priority Review, shortening the time from the standard 10 months to within six months.

At the same time the FDA is reviewing a companion diagnostic test, and the European Medicines Agency is also reviewing the drug in these indications along with other regulators.

In the phase 3 RATIFY trial which investigated midostaurin plus standard chemotherapy versus placebo plus standard chemotherapy in adult patients less than 60 years of age with FLT3-mutated AML, those in the midostaurin arm experienced a statistically significant improvement in overall survival with a 23% reduction in risk of death compared to the placebo arm.

The FDA granted midostaurin Breakthrough Therapy designation for the drug earlier this year for newly-diagnosed FLT3-mutated AML.

There were no statistically significant difference in the overall rate of grade 3 or higher haematologic and non-haematologic adverse events in the midostaurin group versus placebo.

The most frequent all-grade adverse events were febrile neutropaenia, nausea, exfoliative dermatitis, vomiting, headache, small red skin spots and fever.

The systemic mastocytosis filing was backed by data from a phase 2 single-arm study.

PKC412 (midostaurin) is an oral, multi-targeted kinase inhibitor in development for the treatment of patients with AML with a FLT3 mutation and for patients with advanced SM.

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