Syndax Pharmaceuticals announced on October 24 2025 that the U.S. Food and Drug Administration has approved Revuforj (revumenib) for the treatment of relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients one year and older. The approval, issued through a supplemental New Drug Application, follows Revuforj’s 2024 approval for R/R acute leukemia with a KMT2A translocation, making it the first and only menin inhibitor approved for two distinct acute leukemia subtypes.
The approval is based on efficacy data from the Phase 2 portion of the pivotal AUGMENT‑101 trial. In 65 patients with R/R NPM1‑mutated AML, the composite complete remission (CR) plus CR with partial hematologic recovery (CRh) rate was 23% (15/65), with a median time to response of 2.8 months and a median duration of CR/CRh of 4.5 months. The safety profile was consistent with prior studies, with differentiation syndrome occurring in 25% of patients and manageable QTc prolongation in 36%.
By adding NPM1‑mutated AML to its label, Syndax expands Revuforj’s addressable market to an additional 30% of AML patients, potentially increasing U.S. sales to a multi‑billion‑dollar opportunity. The first‑in‑class status and the drug’s oral administration position Revuforj as a compelling alternative to existing multi‑agent regimens, strengthening Syndax’s commercial pipeline and reinforcing its leadership in targeted leukemia therapies.
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