FDA Approves Revuforj, a First-in-Class Menin Inhibitor, for Relapsed or Refractory Acute Leukemia with KMT2A Translocation

FDA Approval:
The FDA has approved Revuforj (revumenib), a first-in-class menin inhibitor, for the treatment of relapsed or refractory acute leukemia with a KMT2A translocation in adult and pediatric patients aged 1 year and older[1][2].

Clinical Trial Results:
The approval is based on the AUGMENT-101 clinical trial, which showed robust and durable rates of remission in patients with R/R acute leukemia with a KMT2A translocation. The trial reported a complete remission (CR) plus CR with partial hematological recovery (CRh) rate of 21.2% and a median duration of response of 6.4 months[1][4].

Availability:
The 110 mg and 160 mg tablets of Revuforj are expected to be available through specialty distributors and specialty pharmacies in November 2024. The 25 mg tablets, for patients weighing less than 40 kg, will be commercially available in late Q1 or early Q2 2025. Until then, an oral solution will be available through an expanded access program[1][2].

Pricing:
Revuforj is priced at $39,500 per month, or $474,000 annually before any rebates or discounts[1].

Future Development:
Syndax plans to expand the development of Revuforj across the treatment continuum for KMT2A-rearranged acute leukemias and mutant NPM1 AML, with additional studies and potential future FDA submissions[1][2].

Support Program:
Syndax has established SyndAccess, a program offering personalized support and resources to U.S. patients prescribed Revuforj, including financial assistance for eligible patients[2].