Syndax Pharmaceuticals (NASDAQ: SNDX) has announced that the U.S. Food and Drug Administration (FDA) 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. This approval marks the second indication for Revuforj in less than a year, further solidifying Syndax’s leadership in menin inhibition.
Revuforj had previously received FDA approval in 2024 for the treatment of R/R acute leukemia with a kmt2a translocation in adult and pediatric patients one year and older. With this new approval, Revuforj becomes the first and only FDA-approved therapy for both R/R AML with an NPM1 mutation and R/R acute leukemia with a kmt2a translocation.
The expansion of the Revuforj label is based on efficacy data from patients with R/R NPM1 mutated AML in the phase 2 portion of the pivotal augment-101 trial. The rate of complete remission (CR) plus CR with partial hematological recovery (CRh) was 23% (15/65 pts; 95% CI: 14%, 35%). The median time to CR or CRh response was 2.8 months, and the median duration of CR or CRh was 4.5 months. Results from the augment-101 trial were published in the journal Blood and presented at the 2025 European Hematology Association (EHA) Annual Congress Meeting.
The safety evaluation of Revuforj was based on the FDA’s analysis of 241 patients (207 adult and 34 pediatric patients) with R/R acute leukemia with an NPM1 mutation or a kmt2a translocation who were treated with Revuforj in clinical trials. The most common adverse reactions are consistent with the known safety profile of Revuforj.
Revumenib was added to the National Comprehensive Cancer Network® (NCCN®) Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for AML as a category 2a recommended treatment option for R/R NPM1m AML based on published data from the augment-101 trial. Revumenib is also included in the NCCN guidelines for AML and acute lymphoblastic leukemia (ALL) as a category 2a recommended treatment option for R/R acute leukemia with a kmt2a rearrangement.
Revuforj is available for order in the United States through Syndax’s existing network of specialty distributors and specialty pharmacies. Syndax has established SyndAccess®, a comprehensive program that offers personalized support and resources to U.S. patients who are prescribed Revuforj, including financial assistance for eligible patients. As a result of these announcements, the company's shares have moved -2.85% on the market, and are now trading at a price of $14.68. For more information, read the company's full 8-K submission here.
