FDA Expands Revumenib Approval to NPM1-Mutant Relapsed/Refractory AML in Patients 1 and Older

The decision follows a priority review supported by AUGMENT-101 results demonstrating meaningful remission in a heavily pretreated NPM1-mutant population.

Overview

The new indication covers adult and pediatric patients at least 1 year old with relapsed or refractory AML harboring a susceptible NPM1 mutation who lack satisfactory alternatives.
In AUGMENT-101, the complete remission or complete remission with partial hematologic recovery rate was 23.1% with a median duration of 4.5 months.
Expanded cohort data presented at EHA showed a 26.0% CR/CRh rate, a 32.5% composite complete response rate, an overall response rate of 48.1%, and markedly longer survival for responders.
Median overall survival for the cohort was 4.8 months, while patients who achieved CR/CRh had a median overall survival of 23.3 months.
The approval follows a supplemental NDA submitted June 25, 2025 under priority review and builds on revumenib’s November 2024 approval for KMT2A-rearranged disease.