FDA Approves Syndax's Revuforj for NPM1 Mutated AML

By Tamara Parker • Monday, October 27 10:26 • 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, further solidifying Syndax’s leadership in menin inhibition.

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.

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, and 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.

This approval further establishes Revuforj as a significant option for patients with r/r AML and reinforces Syndax’s commitment to providing innovative cancer therapies. As a result of these announcements, the company's shares have moved -7.83% on the market, and are now trading at a price of $13.53. Check out the company's full 8-K submission here.

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