FDA Grants Approval to Revumenib for Patients With R/R NPM1-Mutated Acute Myeloid Leukemia
On October 24, 2025, the US Food and Drug Administration (FDA) approved revumenib, a menin inhibitor, for adult and pediatric patients 1 year and older with relapsed or refractory (R/R) acute myeloid leukemia (AML) who have a nucleophosmin 1 (NPM1) mutation without any satisfactory alternative treatment options.
Approval was based on results from AUGMENT-101 (SNDX-5613-0700; NCT04065399), an open-label, multicenter, single-arm trial evaluating the safety and efficacy of revumenib among patients with R/R AML. Eligible patients were required to have a susceptible NPM1 mutation confirmed by next-generation sequencing or polymerase chain reaction of the last exon of NPM1. Efficacy was determined by the rates of complete remission (CR), complete remission with partial hematologic recovery (CRh), duration of CR and CRh, and conversion from transfusion dependence to transfusion independence.
Revumenib therapy was administered once every 12 hours, at 163 mg (95 mg/m2 if weight < 40 kg) in addition to a strong cytochrome P450 inhibitor, in 28-day cycles. The efficacy population included 94 treated patients, of which 46 were red blood cell (RBC) and/or platelet transfusion-dependent.
The CR+CRh rate was 23.1% (95% CI, 13.5–35.2), with a median duration of 4.5 months (95% CI, 1.2–8.1). Among those RBC and/or platelet transfusion-dependent at baseline, 17% achieved transfusion independence during any 56-day period following treatment initiation.
The prescribing information includes warnings and precautions for differentiation syndrome, QTc interval prolongation, and Torsades de Pointes, as well as embryo-fetal toxicity. The recommended dosage of revumenib differs according to patient weight and concomitant use of strong CYP3A4 inhibitors.
Source:
US Food and Drug Administration. Accessed October 24, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-revumenib-relapsed-or-refractory-acute-myeloid-leukemia-susceptible-npm1-mutation
