FDA Approves Revumenib: A New Hope for AML with NPM1 Mutation

On October 24, 2025, the U.S. Food and Drug Administration approved Revumenib (Revuforj, Syndax Pharmaceuticals, Inc.), a menin inhibitor, for adult and pediatric patients aged 1 year and older with relapsed or refractory acute myeloid leukemia (AML) having a susceptible nucleophosmin 1 (NPM1) mutation and no satisfactory alternative treatments.

The approval was based on the AUGMENT-101 trial (NCT04065399), a multicenter open-label study confirming NPM1 mutation by advanced genetic testing. The main efficacy outcomes included the rates of complete remission (CR) complete remission 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% to 35.2%), with a median duration of 4.5 months (95% CI: 1.2 to 8.1 months). Importantly, among 46 patients who were dependent on red blood cell and/or platelet transfusions at baseline, 8 patients (17%) became independent of transfusions during any 56-day period following treatment.

Revumenib dosage is tailored to patient weight and concurrent use of certain enzyme inhibitors. The prescribing information includes warnings for differentiation syndrome, QTc prolongation, torsades de pointes, and embryo-fetal toxicity.

This approval marks a major advance for this AML subtype, offering a targeted treatment option with the potential to improve remission rates and reduce transfusion needs.

This development adds to the portfolio of menin inhibitors and represents hope for patients with limited options. Clinicians are encouraged to review updated guidelines and discuss treatment implications with patients.

Learn more: FDA approves revumenib for relapsed or refractory acute myeloid leukemia with a susceptible NPM1 mutation