Syndax Pharmaceuticals (SNDX) FDA Announcement summary

Introduction and purpose

• Revuforj (revumenib) received FDA approval as the first and only menin inhibitor for relapsed or refractory acute leukemia with KMT2A translocation in adults and children aged one year and older.

• The approval addresses a significant unmet need in a population with poor prognosis and high relapse rates.

• Approval followed Breakthrough Therapy, Fast Track, Orphan Drug, and Priority Review designations, and was reviewed under the Real-Time Oncology Review Program.

Details of approval or decision

• Revuforj is indicated for relapsed or refractory acute leukemia with KMT2A translocation in adults and children, available in 25mg, 110mg, and 160mg tablets; 25mg tablets expected late Q1 or early Q2 2025, with oral solution accessible via expanded access until then.

• Approval was based on data from the pivotal AUGMENT-101 Phase I/II trial, showing robust and durable remission rates.

• The product is positioned for future label expansion and broader franchise opportunities.

Impact on industry and stakeholders

• Revuforj addresses a significant unmet need in acute leukemia, especially for younger adults and children with poor prognosis.

• The initial U.S. market opportunity is estimated at 2,000 patients and a $750 million addressable market, with potential expansion to a >$4B market.

• The launch is expected to secure first-mover advantage in a new therapeutic class, with a broad label covering both adult and pediatric populations.

• Robust patient support programs and experienced launch teams are in place to ensure access and engagement.