FDA Approves Axatilimab-Csfr for Chronic Graft-Versus-Host Disease

The American Society for Transplantation and Cellular Therapy (ASTCT) applauds the U.S. Food and Drug Administration's (FDA) approval of axatilimab-csfr, marketed as Niktimvo by Incyte Corporation. This new therapy addresses chronic graft-versus-host disease (cGVHD), a severe condition affecting patients post-allogeneic stem cell transplantation.

Efficacy was evaluated in the AGAVE-201 study, a trial involving adult and pediatric patients with recurrent or refractory cGVHD. The results showed potential improvements in patient outcomes, highlighting the drug's effectiveness after other treatments had failed.

Corey Cutler, MD, MPH, ASTCT President and co-first author of AGAVE-201, emphasized the significance of this approval. He highlighted the strong evidence from the study and noted the urgent need for new treatments in this area.

The FDA's approval process was facilitated by the Assessment Aid, allowing for efficient review. Niktimvo received priority review, Orphan Drug Designation, and Fast Track Designation, marking its importance in fulfilling a significant unmet medical need.

ASTCT continues to support advancements in transplantation and cellular therapy through research and advocacy, viewing the approval of Niktimvo as a pivotal moment for cGVHD patients.