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news.The US Food and Drug Administration (FDA) has accepted under priority review Novartis' new drug application (NDA) seeking approval of breakthrough therapy-designated PKC412 (midostaurin) investigational, oral, multi-targeted kinase inhibitor to treat acute myeloid leukemia (AML).
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PKC412 is indicated to treat AML in newly-diagnosed adults with an FMS-like tyrosine kinase-3 (FLT3) mutation, as well as for the treatment of advanced systemic mastocytosis (SM).
The NDA is based on the phase III RATIFY trial in AML and a single-arm phase II study of patients with SM.
In the RATIFY trial, the addition of midostaurin to standard chemotherapy reduced the risk of death by 23% compared to the placebo arm in FLT3-mutated AML patients.
The phase II data submitted for midostaurin in SM demonstrated that the drug had an overall response rate of 60% and a median duration of response of 24.1 months.
The median overall survival was 28.7 months. Low-grade nausea, vomiting, and diarrhea were the most common adverse events.
Grade 3/4 neutropenia, anemia, and thrombocytopenia were mostly reported in patients who had pre-existing cytopenias.
Novartis Oncology CEO Bruno Strigini said: "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."
Novartis said the safety and efficacy profile of PKC412 has not been fully established, and it is not approved for any indication in any market at this time. The company did not give guarantee that the product will become commercially available.
Image: Novartis headquarters in Basel. Photo: courtesy of –Andrew- from Flickr.