Menin Inhibitor Revumenib Under FDA Priority Review for Relapsed or Refractory NPM1-Mutated AML

Revumenib has received FDA Priority Review for its supplemental New Drug Application (NDA) for the treatment of relapsed or refractory, NPM1-mutated acute myeloid leukemia, according to a press release from Syndax Pharmaceuticals, the developer of the drug.

The FDA is reviewing the supplemental NDA under its Real-Time Oncology Review program, with a Prescription Drug User Fee Act target action date of October 25, 2025.

The oral, first-in-class menin inhibitor previously received FDA approval in November 2024 for the treatment of relapsed or refractory acute leukemia with a KMT2A translocation.

The NDA submission is based on the phase 2 AUGMENT-101 trial published in Blood. Updated results were presented at the European Hematology Association 2025 Congress, in which revumenib continued to demonstrate “clinically meaningful responses” in heavily pretreated patients.

Of 77 efficacy-evaluable patients, the rate of complete remission (CR) or CR with partial hematologic recovery (CR+CRh) was 26.0% (95% CI, 16.6%-37.2%), the overall response rate was 48.1% (95% CI, 36.5%-59.7%), and the median duration of CR+CRh was 4.7 months (95% CI, 2.1-8.2). Of the 19 patients who achieved CR+CRh with available measurable residual disease (MRD) status, 12 (63.2%) were MRD negative according to findings from polymerase chain reaction or flow cytometry.

Sixty-six patients (78.6%) experienced a treatment-related adverse event (TRAE). Fifty patients (59.5%) experienced a grade 3 or higher TRAE, most commonly QTc prolongation, anemia, febrile neutropenia, differentiation syndrome, and decreased platelet count.

Read more: Menin Inhibitor Shows Activity in Acute Leukemias