Regulatory approval

Published by the Food and Drug Administration. Approved

The U.S. Food and Drug Administration (FDA) granted approval to revumenib for the treatment of adult and pediatric patients 1 year and older with relapsed or refractory acute myeloid leukemia and a susceptible nucleophosmin 1 (NPM1) variant. The approval defines susceptible NPM1 mutations as those that result in a loss of the nucleolar localization signal and the insertion of a new nuclear export signal leading to the accumulation of mutant NPM1 in the cytoplasm of AML cells; the most common of such NPM1 variants in patients with AML being Types A (c.860_863dupTCTG), B (c.863_864insCATG), and D (c.863_864insCCTG).

This is written in the approval document as:

REVUFORJ is a menin inhibitor indicated for the treatment of relapsed or refractory acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients 1 year and older who have no satisfactory alternative treatment options.

Citation

Syndax Pharmaceuticals, Inc. Revuforj (revumenib) [package insert]. U.S. Food and Drug Administration website. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/218944s003lbl.pdf. Revised October 2025. Accessed December 26, 2025.

Therapeutic response

Precision oncology relationships for therapeutic response derived from this regulatory approval.

Organizations	Biomarkers	Cancer types	Therapies
FDA (2)	NPM1 p.W288Cfs*12	Acute Myeloid Leukemia	Revumenib

Contributions

Date	Contributor	Description
2025-12-26	Van Allen lab	On 2025-10-24, the US FDA granted approval to revumenib for the treatment of adult and pediatric patients 1 year or older who have no satisfactory alternative treatment options with relapsed or refractory acute myeloid leukemia and a susceptible NPM1 variant.
2025-10-24	Food and Drug Administration	Indication received traditional approval.