FDA approves axatilimab for the treatment of cGvHD

On August 14, 2024, the U.S. Food and Drug Administration (FDA) granted approval to axatilimab, a colony stimulating factor-1 receptor-blocking antibody, for the treatment of chronic graft-versus-host disease (cGvHD) after ≥2 prior lines of systemic therapy in both adult and pediatric patients who weigh ≥40 kg.¹

This approval was based on data from the phase II, randomized, open-label, multicenter AGAVE-201 trial (NCT04710576), which evaluated axatilimab at 3 different dose levels in patients with recurrent or refractory cGvHD.¹

Pivotal data from AGAVE-201¹

• In total, 79 patients were treated at the final recommended dose of 0.3 mg/kg to a maximum dose of 35 mg, as an infusion every 2 weeks until progression or toxicity deemed unacceptable.
• The overall response rate at the recommended dose was 75%.
• The median time to first response was 1.5 months and the median duration of response was 1.9 months.
• The most common adverse events included increased aspartate aminotransferase, infections, and increased alanine aminotransferase.