FDA approves revumenib for relapsed or refractory acute myeloid leukemia with a susceptible NPM1 mutation
Regulatory approval: On Oct 24, 2025, the FDA approved revumenib (Revuforj; Syndax), a menin inhibitor, for adults and pediatric patients ≥1 year with relapsed or refractory AML harboring a susceptible NPM1 mutation who have no satisfactory alternative treatment options. Approval was supported by a single-arm cohort of the open-label, multicenter AUGMENT-101 trial (NCT04065399), with a CR+CRh rate of 23.1% and median CR+CRh duration of 4.5 months.
FDA approval (Oct 24, 2025)
The FDA approved revumenib (Revuforj; Syndax Pharmaceuticals), a menin inhibitor, for relapsed or refractory acute myeloid leukemia (AML) with a susceptible NPM1 mutation in adult and pediatric patients aged ≥1 year who have no satisfactory alternative treatment options.
Supporting evidence
Efficacy and safety were evaluated in a single-arm cohort of an open-label, multicenter trial: SNDX-5613-0700 (AUGMENT-101; NCT04065399). Susceptible NPM1 mutation status was confirmed using next-generation sequencing or PCR of the last exon of NPM1.
Primary efficacy outcomes included complete remission (CR), CR with partial hematologic recovery (CRh), duration of CR+CRh, and conversion from transfusion dependence to independence.
- CR+CRh rate: 23.1% (95% CI, 13.5–35.2)
- Median CR+CRh duration: 4.5 months (95% CI, 1.2–8.1)
- Among 46 patients transfusion-dependent at baseline, 8 (17%) became independent of RBC and platelet transfusions during any 56-day post-baseline period.
Key safety warnings/precautions
Prescribing information includes warnings and precautions for differentiation syndrome, QTc prolongation and Torsades de Pointes, and embryo-fetal toxicity.
Regulatory notes
The FDA review used Real-Time Oncology Review (RTOR) and the application received priority review; revumenib has fast track designation. Full prescribing information is expected on Drugs@FDA.