In a significant advancement for cancer treatment, the US Food and Drug Administration (FDA) has approved Syndax Pharmaceuticals' Revuforj (revumenib) for the treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A (KMT2A) gene translocation. This approval marks Revuforj as the first and only menin inhibitor available for this specific type of leukemia, offering new hope for both adult and pediatric patients.

The approval was based on positive results from the AUGMENT-101 clinical trial, which demonstrated robust and durable remission rates in patients with KMT2A translocation. The trial involved 104 patients, with a complete remission (CR) plus CR with partial hematological recovery (CRh) rate of 21.2%. The median duration of CR+CRh was 6.4 months, and the median time to achieve CR or CRh was 1.9 months.

Revuforj's approval was facilitated by the FDA's Real Time Oncology Review (RTOR) program, highlighting the drug's potential to significantly improve outcomes for patients with this aggressive form of leukemia. The FDA had previously granted Revuforj Breakthrough Therapy and Fast Track designations, underscoring its importance in the treatment landscape.

Syndax Pharmaceuticals, headquartered in Waltham, Massachusetts, is preparing to launch Revuforj in the US market this month. The company has also established SyndAccess™, a program designed to support patients and provide financial assistance to those eligible.

The safety profile of Revuforj was consistent with previous findings, with common adverse reactions including hemorrhage, nausea, and musculoskeletal pain. Despite these, the drug's potential benefits in treating a condition with historically poor prognosis are significant.

Syndax is also exploring the use of Revuforj in other leukemia subtypes, including NPM1-mutant acute myeloid leukemia, with ongoing trials expected to provide further insights into its efficacy.

This FDA approval represents a major milestone for Syndax and a promising new option for patients battling relapsed or refractory acute leukemia with KMT2A translocation.