Syndax Pharmaceuticals宣布，FDA已对Revuforj治疗复发性或难治性mNPM1急性骨髓性白血病的补充新药申请进行优先审查

 PDUFA action date set for October 25, 2025 –

– sNDA being reviewed under FDA's RTOR program –

– Revumenib has the potential to become the first and only menin inhibitor approved in both R/R mNPM1 AML and R/R KMT2Ar acute leukemia –

 Syndax Pharmaceuticals (NASDAQ:SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review for its supplemental New Drug Application (sNDA) for Revuforj® (revumenib) for the treatment of relapsed or refractory (R/R) mutant NPM1 (mNPM1) acute myeloid leukemia (AML). The sNDA is being reviewed under the FDA's Real-Time Oncology Review (RTOR) program and has been assigned a Prescription Drug User Fee Act (PDUFA) target action date of October 25, 2025. RTOR allows for a more efficient review and close engagement between the sponsor and the FDA throughout the submission process.

"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," said Michael A. Metzger, Chief Executive Officer. "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, first-in-class menin inhibitor that was FDA approved in 2024 for the treatment of R/R acute leukemia with a KMT2A translocation in adult and pediatric patients one year and older. The sNDA, if approved, would expand the indication for Revuforj to include patients with R/R AML who have an NPM1 mutation, the most common genetic alteration in AML. The sNDA is supported by positive pivotal data from the AUGMENT-101 trial of revumenib in patients with R/R mNPM1 AML. Results from this population were published in the journal Blood in May 2025 and presented at the European Hematology Association (EHA) Annual Congress Meeting in June 2025.

About Mutant NPM1 (mNPM1) Acute Myeloid Leukemia (AML)

Mutations in the NPM1 gene are the most common genetic alteration observed in adults with AML, occurring in approximately 30% of cases. Mutations in this gene play a critical role in the development of mutant NPM1 (mNPM1) AML, an aggressive blood cancer associated with high rates of relapse. Patients with relapsed or refractory mNPM1 AML have a poor prognosis and critical unmet need. Similar to KMT2A-rearranged acute leukemia, mNPM1 AML is highly dependent on the menin-KMT2A interaction and disruption of this interaction has been shown to lead to downregulation of certain leukemogenic genes. mNPM1 AML is routinely diagnosed through currently available screening techniques. There are currently no approved therapies that selectively target the underlying disease mechanisms driving mNPM1 AML.