Syndax Pharmaceuticals announced that the U.S. FDA has approved its menin inhibitor, the first and only treatment of its kind, for an aggressive form of acute leukemia in adult and pediatric patients one year and older.
Revumenib, branded Revufori, was approved for relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation — a genetic alteration typically associated with a poor prognosis. When introduced, Revufori fits into the binding pocket of menin and displaces KMT2A. In disrupting the binding interaction, the HOX and MEIS genes are turned off and leukemic cell growth is halted.
The efficacy evaluation of Revuforj was based on an FDA analysis of 104 patients with R/R acute leukemia with a KMT2A translocation who were treated with Revuforj in the phase 1/2 AUGMENT-101 trial. In the efficacy population, the rate of complete remission (plus complete remission with partial hematological recovery) was 21%.
The treatment was granted priority review back in March and given a PDUFA date of September 26, 2024. In July, the agency notified Syndax that it needed additional time to conduct a full review of supplemental information, adding three months to the timeline and pushing the PDUFA date to December 26, 2024.
Massachusetts-based Syndax says it is prepared to launch Revuforj this month, and expects that the 110 and 160 mg tablets will be available for order in the U.S. through a network of specialty distributors and specialty pharmacies in November. The 25 mg tablets, which may be used to treat patients who weigh less than 88 lbs., will be commercially available in late first quarter or early second quarter of 2025.
Syndax is also developing Revuforj across the treatment continuum for KMT2A-rearranged acute leukemias and mutant NPM1 acute myeloid leukemia.