On October 24, 2025, the U.S. Food and Drug Administration (FDA) approved revumenib (Revuforj), a menin inhibitor, for the treatment of relapsed or refractory acute myeloid leukemia with a susceptible nucleophosmin 1 (NPM1) mutation, in adult and pediatric patients 1 year and older who have no satisfactory alternative treatment options.
AUGMENT-101
Efficacy was evaluated in a single-arm cohort of an open-label, multicenter trial (SNDX-5613-0700/AUGMENT-101; ClinicalTrials.gov identifier NCT04065399). A susceptible NPM1 mutation was confirmed in all enrolled patients by using next-generation sequencing or polymerase chain reaction to look at the last exon of NPM1.
The main efficacy outcome measures were complete remission rate (CR), CR with partial hematologic recovery (CRh), CR+CRh duration, and rate of conversion from transfusion dependence to transfusion independence. The CR+CRh rate was 23.1% (95% confidence interval [CI] = 13.5%–35.2%), and the median CR+CRh duration was 4.5 months (95% CI = 1.2–8.1 months). Of the 46 patients dependent on red blood cell and/or platelet transfusions at baseline, 8 (17%) became independent of red blood cell and platelet transfusions during any 56-day postbaseline period.
The prescribing information includes warnings and precautions for differentiation syndrome, QTc interval prolongation and torsades de pointes, and embryofetal toxicity.
Recommended Dosage
The recommended revumenib dose varies by patient weight and concomitant use of strong CYP3A4 inhibitors. See the prescribing information for specific dosage information.
Expedited Programs
This review used the Real-Time Oncology Review program, which streamlined data submission prior to the filing of the entire clinical application, and the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.
This application was granted Priority Review and Fast Track designations.
