The FDA has granted approval to Revuforj (revumenib) for the treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A (KMT2A) gene translocation in adult and pediatric patients one year and older. Developed by Syndax Pharmaceuticals, Revuforj is a menin inhibitor and represents the first therapy of its kind to be approved by the FDA for this indication.
The approval was primarily based on data from the AUGMENT-101 clinical trial, which demonstrated the drug's efficacy in treating this patient population. Syndax plans to make Revuforj, available in 110 mg and 160 mg tablets, through specialty distributors starting in November.
"The approval of Revuforj is a remarkable achievement," said Michael A. Metzger, CEO of Syndax, in a press release. He further acknowledged the contributions of patients, clinicians, and the Syndax team. "We are well-prepared to launch Revuforj this month and we are committed to rapidly advancing the development of Revuforj across the treatment continuum for KMT2A-rearranged acute leukemias and mutant NPM1 AML."
Ghayas C. Issa, MD, from The University of Texas MD Anderson Cancer Center, emphasized the significance of this approval: "The FDA approval of the first menin inhibitor is a major breakthrough for patients with R/R acute leukemia with a KMT2A translocation, a genetic alteration associated with a very poor prognosis. The significant clinical benefit and robust efficacy seen with Revuforj represents a substantial improvement over what has been historically observed in these patients with previously available therapies and has the potential to be an important new treatment option for patients."
Background on Revumenib and Syndax
In July 2024, the FDA extended the Prescription Drug User Fee Act (PDUFA) action date for revumenib's New Drug Application (NDA) to December. At that time, Metzger stated, "Revumenib, upon approval, will be the first drug indicated to treat patients with KMT2A-rearranged acute leukemia, a population with significant unmet need. We are confident that the data from the AUGMENT-101 trial, as well as the additional information provided to the FDA, support approval and continue to demonstrate the meaningful benefit revumenib brings to patients with this devastating disease."
Revuforj is part of Syndax's broader pipeline of oncology medications. Earlier in November, Syndax entered into a $350 million royalty funding agreement with Royalty Pharma for Niktimvo. This agreement is expected to fund Syndax through profitability, while allowing them to retain profits from Niktimvo and its future growth potential.
