Syndax Announces FDA Approval of Revuforj® (revumenib) in Adult and Pediatric Patients with Relapsed or Refractory NPM1 Mutated Acute Myeloid Leukemia

Core Insights - Syndax Pharmaceuticals has received FDA approval for Revuforj (revumenib) as the first and only therapy for both relapsed or refractory (R/R) acute myeloid leukemia (AML) with an NPM1 mutation and R/R acute leukemia with a KMT2A translocation, marking a significant advancement in treatment options for these aggressive blood cancers [2][3][4] Company Developments - Revuforj is now approved for use in adult and pediatric patients aged one year and older who have no satisfactory alternative treatment options, expanding its indication from a previous approval for R/R acute leukemia with a KMT2A translocation [2][11] - The approval is based on data from the Phase 2 portion of the AUGMENT-101 trial, which demonstrated a complete remission (CR) plus CR with partial hematological recovery (CRh) rate of 23% among patients with R/R NPM1 mutated AML [3][4] - The median time to CR or CRh response was reported at 2.8 months, with a median duration of 4.5 months [3] Industry Impact - The inclusion of Revuforj in the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines as a category 2A recommended treatment option for R/R NPM1 mutated AML and R/R acute leukemia with a KMT2A rearrangement underscores its significance in the oncology landscape [5][6] - The approval of Revuforj is expected to set a new standard of care for patients with these difficult-to-treat blood cancers, as highlighted by experts in the field [4][5] - The company is committed to supporting patient access through its SyndAccess program, which provides personalized support and financial assistance for eligible patients [8]