FDA Approves Syndax Pharmaceuticals' Revuforj for R/R AML

By Tamara Parker • Saturday, October 25 10:21 • Permanent Link

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 who have no satisfactory alternative treatment options. This marks the second approved indication for Revuforj in less than one year. 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 patients; 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. Revuforj is 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 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.

Revuforj was added to the National Comprehensive Cancer Network® (NCCN®) Clinical Practice Guidelines in Oncology for AML as a category 2A recommended treatment option for R/R NPM1m AML based on published data from the AUGMENT-101 trial. Revuforj 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.

The most common adverse reactions are consistent with the known safety profile of Revuforj. Revuforj is available for order in the United States through Syndax’s existing network of specialty distributors and specialty pharmacies. As part of its commitment to supporting patients and removing barriers to access, 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. Following these announcements, the company's shares moved -2.85%, and are now trading at a price of $14.68. Check out the company's full 8-K submission here.

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