University Hospital Regensburg

FDA approved axatilimab for chronic GVHD, targeting CSF-1 receptor. Dr. Wolff highlights its high response rate in heavily pretreated patients and unique side effect profile. Future trials aim to evaluate axatilimab as a primary treatment, potentially reducing reliance on steroids.

The FDA approved Niktimvo (axatilimab) for chronic graft-versus-host disease (GVHD) in patients who have progressed on at least 2 prior systemic therapies. GVHD can be acute or chronic, with chronic affecting more organs and requiring longer-term treatment. Current prevention measures, including calcineurin inhibitors and methotrexate, have reduced chronic GVHD incidence to around 30-35%, with new developments like the Orca-T platform potentially lowering it further to 5-10%. However, complete elimination of GVHD is unrealistic, necessitating ongoing treatment options.

Niktimvo™ (axatilimab-csfr) met primary endpoint in AGAVE-201 trial, showing 74% response rate in 0.3 mg/kg dose cohort for chronic graft-versus-host disease (GVHD). FDA approved Niktimvo for GVHD treatment after two prior therapies, and it's added to NCCN Guidelines as a category 2A recommendation.