Syndax Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has approved Revuforj® (revumenib) as the first and only menin inhibitor for the treatment of relapsed or refractory (r/r) acute leukemia with a lysine methyltransferase 2a gene (kmt2a) translocation in adult and pediatric patients one year and older. The FDA approval was based on positive data from the Augment-101 clinical trial, where Revuforj delivered robust and durable rates of remission in r/r acute leukemia patients with a kmt2a translocation.
In the efficacy population, the rate of complete remission (CR) plus CR with partial hematological recovery (CRh) was 21% (22/104 patients; 95% CI: 13.8%, 30.3%). The median duration of CR+CRh was 6.4 months (95% CI: 2.7, not estimable) and the median time to CR or CRh was 1.9 months (range: 0.9, 5.6 months). Additionally, 23% (24/104 patients) of patients underwent hematopoietic stem cell transplantation (HSCT) following treatment with Revuforj.
The safety evaluation of Revuforj was based on an FDA analysis of 135 patients with r/r acute leukemia with a kmt2a translocation who were treated with Revuforj. The most common adverse reactions (≥20%) including laboratory abnormalities were hemorrhage, nausea, phosphate increased, musculoskeletal pain, infection, aspartate aminotransferase increased, febrile neutropenia, alanine aminotransferase increased, parathyroid hormone intact increased, bacterial infection, diarrhea, differentiation syndrome, electrocardiogram QT prolonged, phosphate decreased, triglycerides increased, potassium decreased, decreased appetite, constipation, edema, viral infection, fatigue, and alkaline phosphatase increased.
Syndax expects that the 110 and 160 mg tablets of Revuforj will be available for order in the United States through a network of specialty distributors and specialty pharmacies in November. The company also anticipates that the 25 mg tablets, which may be used to treat patients who weigh less than 40 kg, will be commercially available in late first quarter or early second quarter of 2025.
In addition to the approval of Revuforj, Syndax has established SyndAccess™, a 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 1.3% on the market, and are now trading at a price of $16.03. Check out the company's full 8-K submission here.
