Syndax Pharmaceuticals announced a significant stride in cancer therapy with the U.S. Food and Drug Administration (FDA) granting Priority Review to its supplemental New Drug Application (sNDA) for Revuforj® (revumenib). This sNDA specifically targets the treatment of relapsed or refractory (R/R) mutant NPM1 (mNPM1) acute myeloid leukemia (AML), a particularly aggressive blood cancer. This decision fast-tracks the review process, setting a PDUFA (Prescription Drug User Fee Act) target action date for October 25, 2025.

The sNDA is being reviewed under the FDA's Real-Time Oncology Review (RTOR) program, an initiative designed for more efficient and collaborative review of oncology drugs, ensuring that safe and effective treatments reach patients as quickly as possible. This program facilitates close engagement between Syndax and the FDA throughout the submission, allowing for the review of individual sections of the application as they become available.

Revuforj is an oral, first-in-class menin inhibitor that received initial FDA approval in 2024 for the treatment of R/R acute leukemia with a KMT2A translocation in both adult and pediatric patients. If this sNDA is approved, Revuforj would expand its indication to include R/R AML patients with an NPM1 mutation, which is the most common genetic alteration in adult AML, affecting approximately 30% of cases. These patients face a poor prognosis and a critical unmet medical need. Mutations in the NPM1 gene play a pivotal role in the development of mNPM1 AML, and similar to KMT2A-rearranged acute leukemia, this type of AML is highly dependent on the menin-KMT2A interaction. Revuforj works by disrupting this interaction, leading to the downregulation of genes that drive the leukemia. Currently, there are no approved therapies that specifically target this underlying disease mechanism.

The sNDA is supported by compelling positive pivotal data from the AUGMENT-101 trial of revumenib in patients with R/R mNPM1 AML. The promising results from this trial were recently published in the journal Blood in May 2025 and presented at the European Hematology Association (EHA) Annual Congress Meeting in June 2025. This robust data positions Revuforj to potentially become the first and only menin inhibitor approved in both R/R mNPM1 AML and R/R KMT2Ar acute leukemia, solidifying Syndax's leadership in this emerging therapeutic class.

This priority review marks a crucial step forward in addressing the significant unmet need for effective treatments in R/R mNPM1 AML, offering hope for improved outcomes for these patients.