FDA Grants Approval to Axatilimab for Patients With Chronic Graft-Versus-Host Disease

On August 14th, 2024, the US Food and Drug Administration (FDA) granted approval to axatilimab-csfr (axatilimab), a colony stimulating factor-1 receptor-blocking antibody, for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least 2 prior lines of systemic therapy among adult and pediatric patients weighing at least 40 kilograms (kg).

This approval was based on efficacy results from the AGAVE-201 trial. This randomized, open-label, multicenter study investigated 3 dosages of axatilimab-csfr in adult and pediatric patients with recurrent or refractory chronic GVHD who had received at least 2 lines of systemic therapy and required additional treatment. The major efficacy outcome measure was overall response rate (ORR) through cycle 7, day 1; overall response included complete response or partial response according to the 2014 NIH Consensus Development Project on Response Criteria.

Results demonstrated the ORR was 75% (95% CI, 64 to 84) in the 79 patients who were treated with the recommended dosage. The median time to first response was 1.5 months. The median duration of response, which was calculated from first response to progression, death, or new systemic therapies for chronic graft-versus-host disease, was 1.9 months (95% CI, 1.6 to 3.5). Among patients who achieved response, no death or new systemic therapy initiation occurred in 60% (95% CI, 43 to 74) of patients for at least 12 months since response.

It was noted the most common (≥15%) adverse reactions, which included laboratory abnormalities, were increased aspartate aminotransferase, infection (pathogen unspecified), increased alanine aminotransferase, decreased phosphate, decreased hemoglobin, viral infection, increased gamma glutamyl transferase, musculoskeletal pain, increased lipase, fatigue, increased amylase, increased calcium, increased creatine phosphokinase, increased alkaline phosphatase, nausea, headache, diarrhea, cough, bacterial infection, pyrexia, and dyspnea.

Source:

FDA approves axatilimab-csfr for chronic graft-versus-host disease. Press Release. US Food and Drug Administration. Published online August 14, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-axatilimab-csfr-chronic-graft-versus-host-disease