FDA Approves Axatilimab for Chronic Graft-vs-Host Disease

FDA Approves Axatilimab for Chronic Graft-vs-Host Disease

The FDA approved axatilimab-csfr (Niktimvo) for the treatment of adult and pediatric patients weighing at least 40 kg with chronic graft-vs-host disease (cGVHD) whose disease progressed on 2 or more prior lines of systemic therapy.

Of note, axatilimab is a colony-stimulating factor-1 receptor-blocking antibody, according to the FDA’s notice of the approval.

The approval was based on findings from the AGAVE-201 trial (NCT04710576). In this randomized, open-label, multicenter trial, 3 dosages of axatilimab were assessed in adult and pediatric patients with recurrent or refractory cGVHD who previously received at least 2 lines of systemic therapy and needed additional treatment.

The major efficacy outcome of the AGAVE-201 trial was overall response rate (ORR) through Cycle 7 Day 1. During this time, overall response included complete or partial response per the 2014 NIH Consensus Development Project on Response Criteria. The ORR for the 79 patients treated with the recommended dosage of axatilimab was 75% (95% CI, 64%-84%) with a median time to first response of 1.5 months (range, 0.9-5.1).

Researchers also calculated the median duration of response from the first response to progression, death, or new systemic therapies for the treatment of cGVHD. The median duration of response was 1.9 months (95% CI, 1.6-3.5). No death or new systemic therapy initiation occurred for at least 12 months since treatment response in 60% (95% CI, 43%-74%) of patients who achieved a response from axatilimab.

The most common adverse reactions, occurring in at least 15% of patients in the trial, included infection (pathogen unspecified), increased aspartate aminotransferase levels, decreased phosphate levels, increased alanine aminotransferase levels, viral infection, decreased hemoglobin levels, musculoskeletal pain, increased gamma-glutamyl transferase levels, fatigue, increased lipase levels, increased calcium levels, increase amylase levels, increased alkaline phosphatase levels, increased creatine phosphokinase levels, headache, nausea, cough, diarrhea, pyrexia, bacterial infection, and dyspnea.

According to the FDA’s notice, the recommended dose of axatilimab for patients who weigh at least 40 kg is 0.3 mg/kg (maximum dose, 35 mg) administered intravenously over 30 minutes every 2 weeks until unacceptable toxicity or disease progression.

Reference

FDA approves axatilimab-csfr for chronic graft-versus-host disease. FDA. 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