FDA Grants Priority Review To Syndax’s sNDA For Revuforj® (revumenib) in Treatment Of Relapsed Or Refractory mNPM1 Acute Myeloid Leukemia

Syndax Pharmaceuticals, a commercial-stage biopharmaceutical company focused on developing innovative cancer treatments, has announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review for its supplemental New Drug Application (sNDA) for Revuforj® (revumenib). The application seeks approval for the use of Revuforj in treating patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) that carries a mutation in the NPM1 gene (mNPM1), which is the most common genetic mutation in AML. 

The FDA is reviewing the application under its Real-Time Oncology Review (RTOR) program, which is designed to streamline the review process through early and ongoing communication with the sponsor. A Prescription Drug User Fee Act (PDUFA) target action date has been set for October 25, 2025.

Michael A. Metzger, Chief Executive Officer, stated, “We are pleased that the FDA has granted Priority Review to our sNDA in R/R mNPM1 AML, a filing which builds on the initial approval of Revuforj for R/R acute leukemia with a KMT2A translocation in 2024. Syndax is uniquely positioned to continue leading this exciting new therapeutic class with a first- and best-in-class menin inhibitor supported by compelling pivotal data across the broadest population of patients and a strong foundation already established among clinicians, payers, and other key stakeholders.”

Revuforj is an oral menin inhibitor that was approved by the FDA in 2024 for the treatment of R/R acute leukemia with a KMT2A gene translocation in both adults and children aged one year and older. If approved, the new indication would expand the use of Revuforj to include patients with R/R AML and an NPM1 mutation. The sNDA is supported by data from the pivotal AUGMENT-101 trial, which evaluated revumenib in this patient group. The trial results were published in “Blood” in May 2025 and presented at the European Hematology Association (EHA) Annual Congress in June 2025.