FDA Grants Priority Review to Revuforj for Mutant NPM1 AML

The FDA grants priority review to Revuforj’s supplemental new drug application to treat R/R mNPM1 AML: © stock.adobe.com.

The U.S. Food and Drug Administration (FDA) has granted priority review to the supplemental new drug application for Revuforj (revumenib) for the treatment of relapsed or refractory mutant NPM1 acute myeloid leukemia (AML), according to a news release from Syndax.

Revuforj is an oral, first-in-class menin inhibitor used to treat certain types of relapsed or refractory leukemia with specific genetic changes.

The supplemental new drug application is being reviewed through the FDA's real-time oncology review program, which allows for a more efficient review process and close engagement between the agency and the sponsor throughout the submission. The application has been assigned a target action date of October 25, 2025, under the Prescription Drug User Fee Act.

“We are pleased that the FDA has granted priority review to our supplemental new drug application in relapsed or refractory mutant NPM1 AML, a filing which builds on the initial approval of Revuforj for relapsed or refractory acute leukemia with a KMT2A translocation in 2024,” Michael A. Metzger, chief executive officer, said in the news release. “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.”

According to the U.S. FDA website, a priority review designation for a supplemental new drug application is meant to accelerate the evaluation of the drug, with an estimated decision timeline of six months compared with the usual 10. Moreover, this process is specifically reserved for supplemental new drug applications that would potentially represent a meaningful advancement for the treatment of a serious condition compared with current options, if approved.

This potential label expansion is backed by results from the AUGMENT-101 trial, which studied Revuforj in this population. Findings were published in Blood in May 2025 and shared at the European Hematology Association’s annual meeting in June 2025.

Revuforj is an oral treatment that blocks menin and was first approved by the FDA in 2024 for adults and children at least one year old with relapsed or refractory acute leukemia driven by a KMT2A translocation.

Among patients in the AUGMENT-101 trial, 21.2% achieved complete remission plus complete remission with partial hematological recovery with a median duration of 6.4 months. In addition, of the 22 patients who achieved either complete response or complete remission with partial hematological recovery, the median time to either of those outcomes was 1.9 months.

The most common side effects, which occurred in at least 20% of patients, included nausea, bleeding, musculoskeletal pain, high phosphate levels, increased aspartate aminotransferase, infection, febrile neutropenia, increased intact parathyroid hormone, increased alanine aminotransferase, diarrhea, bacterial infection, QT prolongation, differentiation syndrome, increased triglycerides, decreased phosphate, decreased potassium, constipation, decreased appetite, fatigue, viral infection and increased alkaline phosphatase.

The new application now under review aims to expand its use to patients with relapsed or refractory AML who carry an NPM1 mutation, the most common genetic alteration in this disease.

More Information About Revuforj and Mutant NPM1 AML

Mutations in the NPM1 gene, seen in about 30% of adults with AML, define a subtype known as mutant NPM1 AML — an aggressive form of the disease with high relapse rates and limited treatment options, according to the release. These leukemias are strongly dependent on the menin-KMT2A interaction, which helps drive cancer growth by activating specific genes. Disrupting this interaction has shown promise in reducing disease-driving signals. While mutant NPM1 AML can be identified through standard diagnostic methods, no approved treatments currently target its underlying biology, leaving a significant unmet need for patients with relapsed or treatment-resistant disease.

Additional studies of Revuforj, including in newly diagnosed patients, are underway or planned, combining the drug with standard treatments in NPM1-mutated or KMT2A-rearranged leukemia. Revuforj has received several FDA and European designations, including orphan drug, fast track, and breakthrough therapy designations.

Reference

“FDA grants priority review for supplemental application of Revuforj in relapsed or refractory mutant NPM1 acute myeloid leukemia” News release. Syndax Pharmaceuticals, Inc. June 24, 2025.

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