Syndax Pharmaceuticals (NASDAQ: SNDX) has recently 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 approval marks the second indication for Revuforj in less than a year, further solidifying Syndax’s leadership in menin inhibition.
The expansion of the Revuforj label is supported by 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.
Additionally, 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.
AML is a cancer of the bone marrow and blood marked by rapid disease progression. Mutations in the NPM1 gene are the most common genetic alteration observed in AML, occurring in approximately 30% of adults with AML. Mutations in this gene play a critical role in the development of NPM1 mutated AML, an aggressive blood cancer associated with high rates of relapse. Revuforj is available for order in the United States through Syndax’s existing network of specialty distributors and specialty pharmacies.
The approval of Revuforj for the treatment of R/R AML with an NPM1 mutation highlights the compelling and consistent efficacy and tolerability of the therapy in multiple different types of patients. This second indication for Revuforj further underscores its potential as a targeted, oral therapy that is well tolerated and efficacious for these aggressive and difficult-to-treat blood cancers.
Syndax's commitment to supporting patients and removing barriers to access is demonstrated through the establishment of 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 FDA approval reinforces Syndax Pharmaceuticals' position as a commercial-stage biopharmaceutical company advancing innovative cancer therapies and underscores its dedication to innovating for patients with menin-dependent acute leukemias. 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. Check out the company's full 8-K submission here.
