Axatilimab-csfr Approved for Chronic Graftversus- Host Disease

On August 14, 2024, the FDA approved axatilimab-csfr (Niktimvo; Incyte Corporation), a colony-stimulating factor-1 receptor–blocking antibody, for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least 2 prior lines of systemic therapy.

Efficacy was evaluated in AGAVE-201 (NCT04710576), a randomized, open-label, multicenter trial, investigating 3 dosages of axatilimab-csfr in adult and pediatric patients with recurrent or refractory cGVHD 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, where overall response included complete response or partial response according to the 2014 NIH Consensus Development Project on Response Criteria. ORR was 75% (95% CI, 64-84) in the 79 patients treated with the recommended dosage. The median time to first response was 1.5 months (range, 0.9-5.1). The median duration of response (DOR), calculated from first response to progression, death, or new systemic therapies for cGVHD, was 1.9 months (95% CI, 1.6-3.5). In patients who achieved response, no death or new systemic therapy initiation occurred in 60% (95% CI, 43-74) of patients for at least 12 months since response.

The most common adverse reactions, including laboratory abnormalities, were increased aspartate aminotransferase, infection, 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.

The recommended axatilimab-csfr dose in patients who weigh at least 40 kg is 0.3 mg/kg, up to a maximum dose of 35 mg, as an intravenous infusion over 30 minutes every 2 weeks until disease progression or unacceptable toxicity.¹

Editor’s note: This report was based on statements issued by the FDA and pharmaceutical companies mentioned in the approvals.

References

1. Food and Drug Administration. FDA approves axatilimab-csfr for chronic graftversus-host-disease. August 14, 2024. Accessed August 20, 2024. www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-axatilimab-csfr-chronic-graft-versus-host-disease