FDA Approves Revumenib for Relapsed or Refractory Acute Myeloid Leukemia With Susceptible NPM1 Mutation

On October 24, 2025, the FDA approved revumenib (Revuforj) for the treatment of adult and pediatric patients aged 1 years and older with relapsed or refractory acute myeloid leukemia (AML) characterized by a susceptible nucleophosmin 1 (NPM1) mutation with no satisfactory alternative treatment options.¹

The approval was based on results from the AUGMENT-101 trial. The main efficacy outcome measures were complete remission (CR), CR with partial hematological recovery (CRh), CR+CRh duration, and rate of conversion from transfusion dependence to transfusion independence. The CR+CRh rate was 23.1% (95% CI, 13.5-35.2), and the median CR+CRh duration was 4.5 months (95% CI, 1.2-8.1). Of the 46 patients dependent on red blood cell (RBC) and/or platelet transfusions at baseline, 8 (17%) became independent of RBC and platelet transfusions during any 56-day post-baseline period.

Revumenib works as a menin inhibitor, targeting the underlying genetic drivers of AML associated with NPM1 mutations. Prescribing information includes precautions and warnings about differentiation syndrome, QTc interval prolongation and torsades de pointes, and embryo-fetal toxicity.

Reference

1. US Food and Drug Administration. FDA approves revumenib for relapsed or refractory acute myeloid leukemia with susceptible NPM1 mutation. October 24, 2025. Accessed November 6, 2025. www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-revumenib-relapsed-or-refractory-acute-myeloid-leukemia-susceptible-npm1-mutation