14/06/2025
Axatilimab, a high-affinity monoclonal antibody targeting CSF-1R, has demonstrated clinical benefit in patients with chronic graft-versus-host disease (cGVHD). A detailed exposure-response analysis based on data from the AGAVE-201 and SNDX-6352-0503 studies evaluated relationships between axatilimab exposure, efficacy, and safety. Higher exposure was associated with greater treatment response, while lower exposure decreased the odds of improvement. Ten of eleven safety endpoints showed increased risk with higher drug exposure. Among evaluated regimens, 0.3 mg/kg every 2 weeks offered the best benefit–risk profile. Importantly, this analysis formed part of the regulatory filing package that supported axatilimab’s FDA approval for cGVHD, highlighting the critical role of model-informed drug development in regulatory decision-making.
