FDA grants orphan drug designation to axatilimab for the treatment of chronic GvHD

Axatilimab, an anti-colony stimulating factor 1 receptor monoclonal antibody for the treatment of chronic graft-versus-host disease (cGvHD), was granted orphan drug designation by the U.S. Food and Drug Administration (FDA). This designation is given to treatments for rare disorders that affect fewer than 200,000 or for underserved patient populations.

Axatilimab is thought to act by inhibiting monocyte-derived macrophages which play a key role in the fibrotic disease process during cGvHD. Data from the phase I trial of axatilimab was reported at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, and the results of this trial can be found in a recent article on the GvHD Hub, here. The overall response rate in this trial of 15 patients was 57% and the median time to response was 1.9 months (1−11 months). Treatment-emergent adverse events Grades 3−4 were recorded in 47% of patients and involved impaired liver and kidney function as well as pneumonia.

A phase II study (AGAVE-201) is planned to investigate three doses of axatilimab, and topline data is expected in 2023.