FDA Approves Syndax's Revuforj (Revumenib) for Relapsed or Refractory Acute Leukemia with KMT2A Translocation

• The FDA has approved Syndax Pharmaceuticals' Revuforj (revumenib) as the first and only menin inhibitor for relapsed or refractory acute leukemia with KMT2A translocation in patients aged one year and older.
• Revuforj's approval was based on the Phase I/II AUGMENT-101 trial, where 21% of patients achieved complete remission with partial hematological recovery, and 23% underwent stem cell transplantation.
• The drug is expected to be available in the US through specialty distributors in November 2024, with 25mg tablets and an oral solution planned for early 2025.
• Syndax Pharmaceuticals is also advancing the development of Revuforj across the treatment continuum for KMT2A-rearranged acute leukemias and mutant NPM1 AML.

The U.S. Food and Drug Administration (FDA) has granted approval to Syndax Pharmaceuticals' Revuforj (revumenib) for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in patients aged one year and older. This approval marks Revuforj as the first and only menin inhibitor available for this patient population.

Clinical Efficacy and Trial Data

The approval of Revuforj was expedited through the FDA’s Real Time Oncology Review program, following breakthrough therapy, fast track designations, and priority review. The efficacy of Revuforj was evaluated in the Phase I/II AUGMENT-101 trial, which enrolled 104 patients with the specified acute leukemia. In this study, 21% of patients achieved complete remission with partial hematological recovery. The median duration of these responses was 6.4 months, with a median time to response of 1.9 months. Notably, 23% of patients were able to proceed to hematopoietic stem cell transplantation post-treatment.

These results align with the Phase II interim analysis from the same trial, reinforcing the potential of Revuforj in treating this aggressive form of leukemia.

Safety Profile and Availability

The safety profile of Revuforj was assessed in 135 patients as part of the FDA’s analysis for approval. Syndax Pharmaceuticals anticipates that Revuforj 110mg and 160mg tablets will be available through specialty distributors and pharmacies in the US starting in November 2024. The 25mg tablets, designed for patients weighing under 40kg, are expected to be available by early 2025, with an interim oral solution provided through an expanded access program.

Company Statements and Future Development

Syndax Pharmaceuticals CEO Michael Metzger stated, “The approval of Revuforj is a remarkable achievement that reflects the dedication and tenacity of everyone involved, especially the patients and clinicians who participated in our trial and our talented Syndax team. We are well-prepared to launch Revuforj this month and we are committed to rapidly advancing the development of Revuforj across the treatment continuum for KMT2A-rearranged acute leukemias and mutant NPM1 AML.”