The U.S. Food and Drug Administration (FDA) has granted approval for Revuforj® (revumenib), as announced by Syndax Pharmaceuticals This approval allows the treatment of adult and pediatric patients (one year and older) with relapsed or refractory (R/R) acute myeloid leukemia (AML) that harbors a susceptible NPM1 mutation, for whom there are no satisfactory alternative treatment options.

This marks the second FDA approval for Revuforj, which previously received clearance in 2024 for R/R acute leukemia with a KMT2A translocation, solidifying its position as the first and only FDA-approved menin inhibitor for both of these aggressive acute leukemia subtypes in adults and children.

The expanded approval is based on compelling efficacy data from the Phase 2 portion of the pivotal AUGMENT-101 trial, which demonstrated a combined complete remission (CR) and CR with partial hematological recovery (CRh) rate of 23% in R/R NPM1 mutated AML patients, with a median duration of response of 4.5 months. This significant advancement addresses a critical unmet need, as NPM1 mutations are the most common genetic alteration in AML, occurring in approximately 30% of adults, and patients with R/R disease face a poor prognosis. Revuforj, an oral, first-in-class menin inhibitor, is already included in the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) as a Category 2A recommended treatment option for R/R NPM1 mutated AML. The safety profile for the second indication is consistent with the known safety profile of Revuforj, with the most common adverse reactions being manageable. Syndax emphasized its commitment to patient access through its comprehensive support program, SyndAccess®, and is actively working to advance this therapeutic class into the frontline setting for menin-dependent acute leukemias.