FDA Approves Axatilimab-csfr for Chronic GVHD

The US Food and Drug Administration (FDA) has approved axatilimab-csfr for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy, according to a press release from the federal agency.

Axatilimab-csfr, sold under the brand name Niktimvo, is a colony-stimulating factor-1 receptor-blocking antibody co-developed by Incyte Corporation and Syndax Pharmaceuticals.

The approval is based on results of the randomized, open-label, multicenter AGAVE-201 trial investigating three doses of the drug: 0.3 mg/kg every two weeks, 1.0 mg/kg every two weeks, or 3.0 mg/kg every four weeks. The study included adult and pediatric patients with recurrent or refractory chronic GVHD, a complication of hematopoietic stem cell transplantation.

The overall response rate was 75% (95% CI, 64-84) in 79 patients treated with the recommended dose of 0.3 mg/kg. The median time to first response was 1.5 months, and the median duration of response was 1.9 months. For at least 12 months, 60% of patients who achieved a response did not experience death or new systemic therapy initiation.

Serious adverse events (AEs) occurred in 44% of patients, according to a press release from Incyte and Syndax. AEs leading to dose interruption in more than two patients included viral infection, infection (pathogen unspecified), bacterial infection, musculoskeletal pain, and pyrexia.

“Advanced chronic GVHD is characterized by the development of fibrotic tissue across multiple organ systems, including, most commonly, the skin and mucosa, and can be extremely difficult to treat, leading to high rates of morbidity and mortality,” Daniel Wolff, MD, PhD, Head of the GVHD Center at the University Hospital Regensburg and lead investigator of the AGAVE-201 trial, said in the press release. “I am highly encouraged by the robust responses observed across all organs and patient subgroups within the heavily pretreated population enrolled in the AGAVE-201 trial. I look forward to having a new and differentiated treatment option for my patients who need additional therapies to address this very difficult to manage, debilitating disease.”