On October 24, 2025, the Food and Drug Administration approved revumenib a menin inhibitor, for relapsed or refractory acute myeloid leukaemia with a susceptible nucleophosmin 1 (NPM1) mutation in adult and paediatric patients 1 year and older who have no satisfactory alternative treatment options.

Full prescribing information for revumenib will be posted on Drugs@FDA.

Efficacy and Safety

Efficacy was evaluated in a single-arm cohort of an open-label, multicenter trial (SNDX-5613-0700, NCT04065399; AUGMENT-101). A susceptible mutation was confirmed in enrolled patients using next generation sequencing or polymerase chain reaction of the last exon of NPM1.

The main efficacy outcome measures were complete remission rate (CR), CR with partial haematological 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.

The prescribing information includes warnings and precautions for differentiation syndrome, QTc interval prolongation and Torsades de Pointes, and embryo-fetal 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 (RTOR) 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. Revumenib has fast track designation. FDA expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.

Source: FDA