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FDA grants priority review to midostaurin for FLT3–mutated AML, advanced systemic mastocytosis
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The FDA granted priority review to midostaurin for the treatment of acute myeloid leukemia in newly diagnosed adults with a FLT3 mutation and for the treatment of advanced systemic mastocytosis, according to the drug’s manufacturer.
“FLT3–mutated AML and advanced systemic mastocytosis are devastating and rare diseases, with significant unmet needs due to limited existing treatment options,” Bruno Strigini, CEO of Novartis Oncology, said in a press release. “This regulatory designation signifies the importance of midostaurin (PKC412, Novartis) as a potential therapy for these patients who haven't had the benefit of targeted medicines.”
The phase 3 RATIFY trial — designed to evaluate the addition of midostaurin to standard chemotherapy in adults aged younger than 60 years with FLT3–mutated AML — demonstrated superior OS with a 23% reduction in risk for death (HR = 0.77) in the treatment arm compared with the placebo arm.
The researchers found no difference in adverse events between arms.
A phase 2 single-arm study designed to evaluate the efficacy of midostaurin in patients with advanced systemic mastocytosis showed a 60% overall response rate with a median duration of response of 24.1 months and a median OS of 28.7 months.
The most frequent adverse events included nausea, vomiting and diarrhea.
The FDA also accepted the premarket approval application for the midostaurin FLT3 companion diagnostic — which was developed in collaboration with Invivoscribe Technologies.