The US Food and Drug Administration (FDA) has approved revumenib (Revuforj®) in patients with relapsed or refractory NPM1-mutated acute myeloid leukemia (AML). The approval is indicated for both adult and pediatric patients one year and older who have no alternative treatment options.
The therapy has another indication for the treatment of relapsed or refractory acute leukemia with a KMT2A translocation in adult and pediatric patients one year and older. That indication was approved by the FDA in 2024.
Syndax Pharmaceuticals, the manufacturer of revumenib, said that the second indication is the only menin inhibitor FDA-approved for multiple AML subtypes.
“We are thrilled to have secured a second indication for Revuforj, making it the first and only menin inhibitor that is FDA-approved for multiple acute leukemia subtypes in both adults and children. The breadth of the indicated patient population highlights the compelling and consistent efficacy and tolerability of Revuforj in multiple different types of patients,” said Michael A. Metzger, CEO. “Our launch into this second population will greatly benefit from physicians’ already strong familiarity with Revuforj and positive experience treating well over 1,000 patients in clinical trials and nearly one year of commercial use.”
According to the company release, the safety evaluation of revumenib was based on the FDA’s analysis of 241 patients (207 adult and 34 pediatric patients) with relapsed or refractory NPM1-mutated or KTM2A-translocated acute leukemia who were treated with revumenib in clinical trials.
