Syndax Secures FDA Approval For Revuforj® (revumenib) To Treat Adult And Pediatric Patients With Relapsed Or Refractory NPM1-Mutated Acute Myeloid Leukemia

Syndax Pharmaceuticals, a commercial-stage biopharmaceutical company developing innovative cancer therapies, 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 aged one year and older who lack satisfactory alternative treatment options. 

This marks the second FDA approval for Revuforj, which was previously approved in 2024 for treating R/R acute leukemia with a KMT2A translocation in both adult and pediatric patients. With this 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.

Michael A. Metzger, Chief Executive Officer of Syndax, expressed his enthusiasm for the new approval, noting that Revuforj is now the first menin inhibitor approved for multiple acute leukemia subtypes in both adults and children. He highlighted that the expanded indication underscores the consistent efficacy and tolerability of the therapy across a wide range of patients. Metzger added that the company’s experience with physicians who have already treated over 1,000 patients in clinical trials and nearly a year of commercial use will help ensure a strong launch into this new patient population.

He further thanked patients, clinicians, and the Syndax team for their contributions, emphasizing the company’s commitment to advancing treatments for menin-dependent acute leukemias and continuing the development of this promising therapeutic class.

The FDA’s expanded approval is based on data from the Phase 2 portion of the pivotal AUGMENT-101 trial, which evaluated patients with R/R NPM1-mutated AML. The study reported a complete remission (CR) plus CR with partial hematologic recovery (CRh) rate of 23% (15 out of 65 patients; 95% CI: 14%, 35%). The median time to achieve CR or CRh was 2.8 months, and the median duration of response was 4.5 months. The results were published in Blood and presented at the 2025 European Hematology Association (EHA) Annual Congress.

According to Dr. Joshua F. Zeidner, Chief of Leukemia Research at the University of North Carolina’s Lineberger Comprehensive Cancer Center, this approval represents a significant advancement in leukemia treatment. He noted that Revuforj provides, for the first time, a well-tolerated, targeted oral therapy for R/R NPM1-mutated AML and R/R KMT2A-translocated acute leukemia, paving the way for a new standard of care for these difficult-to-treat cancers.

The FDA’s safety assessment of Revuforj was based on data from 241 patients (207 adults and 34 children) with R/R acute leukemia featuring either an NPM1 mutation or KMT2A translocation. The most common adverse reactions were consistent with its previously known safety profile. Lore Gruenbaum, Ph.D., Chief Scientific Officer of Blood Cancer United® (formerly The Leukemia & Lymphoma Society), welcomed the approval, emphasizing that new treatments are urgently needed for patients with NPM1-mutated AML. She noted that Revuforj offers new hope by directly targeting the pathway driving this aggressive form of leukemia.

On September 18, 2025, 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 NPM1-mutated AML, based on the AUGMENT-101 data. Revumenib also continues to be listed in the NCCN Guidelines for AML and acute lymphoblastic leukemia (ALL) as a category 2A recommended option for R/R acute leukemia with a KMT2A rearrangement.

AML is a rapidly progressing cancer of the blood and bone marrow. Mutations in the NPM1 gene, present in about 30% of adults with AML, play a critical role in the disease’s development and are linked to high relapse rates and poor outcomes. Revuforj is now available for order in the United States through Syndax’s established network of specialty distributors and pharmacies. To support patient access, Syndax offers SyndAccess®, a comprehensive patient assistance program that provides financial support and personalized resources to help eligible patients access Revuforj treatment.