FDA Approves Revuforj for NPM1-Mutant Relapsed AML

The FDA approved revumenib (Revuforj) to treat patients aged 1 year and older with relapsed or refractory acute myeloid leukemia (AML) harboring a susceptible NPM1 mutation who have no satisfactory alternative treatment options. The decision was based on data from the open-label, multicenter Phase 2 AUGMENT-101 trial, which showed a 23.1% complete remission/complete remission with partial hematologic recovery (CR/CRh) rate (95% CI, 13.5%–35.2%) and a median remission duration of 4.5 months. In the trial, 17% of transfusion-dependent patients became transfusion-independent during the 56-day post-baseline period. Revuforj, an oral targeted therapy, was previously approved for acute leukemia with KMT2A translocation, giving clinicians a treatment option for two aggressive, genetically defined leukemias. Despite the regulatory milestone, Syndax Pharmaceuticals’ shares fell following the approval announcement.