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Axatilimab is a humanized IgG4 monoclonal antibody that targets the macrophage colony-stimulating factor 1 receptor (CSF-1R) on monocytes and macrophages. By inhibiting CSF-1R, it reduces the levels of pro-inflammatory and pro-fibrotic monocytes and macrophages, thereby addressing both inflammation and fibrosis in chronic graft-versus-host disease (cGVHD). Axatilimab is indicated for adults and pediatric patients weighing at least 40 kg who have failed at least two prior lines of systemic therapy for cGVHD. It is administered as an intravenous infusion. The drug was developed by Syndax Pharmaceuticals, which licensed global rights from UCB. Axatilimab was approved in the United States in August 2024 under the brand name Niktimvo[2][3][5][6][7].
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