FDA approves axatilimab-csfr to treat graft-versus-host disease

FDA approves axatilimab-csfr to treat graft-versus-host disease | Image Credit: © Calin - © Calin - stock.adobe.com.

The FDA has approved axatilimab-csfr (Niktimvo; Incyte Corporation and Syndax Pharmaceuticals) for the treatment of chronic graft-versus-host disease (cGVHD), after failure of at least 2 prior lines of systemic therapy in adult and pediatric patients who weight at least 88 lbs (40 kg), according to a press release from the FDA.

Axatilimab-csfr is a colony stimulating factor-1 receptor-blicking antibody. Efficacy was determined in the AGAVE-201 (NCT04710576) randomized, open-label, multicenter clinical trial that investigated 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," stated the FDA.

ORR was 75% in the 79 patients treated with the recommended dosage (95% CI: 64, 84), with a median time to first response of 1.5 months (95% CI: 1.6, 3.5). No death or new systemic therapy initiation occurred in 60% of patients who achieved response for at least 12 months since response (95% CI: 43, 74).

For patients who weight 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.

The most common adverse reactions (≥ 15%) included: laboratory abnormalities, were increased aspartate aminotransferase (AST), infection (pathogen unspecified), increased alanine aminotransferase (ALT), decreased phosphate, decreased hemoglobin, viral infection, increased gamma glutamyl transferase (GGT), musculoskeletal pain, increased lipase, fatigue, increased amylase, increased calcium, increased creatine phosphokinase (CPK), increased alkaline phosphatase (ALP), nausea, headache, diarrhea, cough, bacterial infection, pyrexia, and dyspnea.

The application for axatilimab-csfr was granted Priority Review by the FDA.

Reference:

FDA approves axatilimab-csfr for chronic graft-versus-host disease. FDA. Press release. August 14, 2024. Accessed August 14, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-axatilimab-csfr-chronic-graft-versus-host-disease