New FDA Approval Issued: Axatilimab-csfr 9-mg, 22-mg Vials for Chronic GVHD

The FDA has approved axatilimab-csfr in 9-mg and 22-mg vial sizes for management of chronic graft-versus-host disease (GVHD) after failure of at least two lines of systemic therapy. The approval is for use of the agent in adult and pediatric patients who weigh at least 40 kg, with dosing at 0.3 mg/kg up to a maximum dose of 35 mg and administered via 30-minute intravenous infusion every two weeks.

The FDA approval was announced in a press release from Incyte and Syndax Pharmaceuticals. The two global biopharmaceutical companies co-develop and commercialize axatilimab-csfr as Niktimvo, a trademark of Incyte. The agent is expected to be released in the US in early February.

“We are thrilled to build on our strong commitment to the GVHD community with the U.S. launch of Niktimvo, a first-in-class therapeutic agent that has demonstrated remarkable responses in patients with chronic GVHD whose response was suboptimal after at least two prior lines of systemic therapy,” commented Incyte Chief Executive Officer Hervé Hoppenot.

Axatilimab-csfr is an antibody that treats chronic GVHD by blocking colony- stimulating factor-1 receptor (CSF-1R), thereby decreasing drivers of fibrosis and inflammation.

“As the first and only FDA-approved anti-CSF-1R antibody targeting the drivers of inflammation and fibrosis in chronic GVHD, Niktimvo represents a major breakthrough for patient care,” remarked Syndax CEO Michael Metzger.

The FDA approval was granted on August 14, 2024, based on impressive data from the AGAVE-201 international clinical trial published in the New England Journal of Medicine. In the trial, axatilimab-csfr, administered at 0.3 mg/kg every two weeks, produced a response at six months in 75% of patients, and the trial’s primary endpoint was met in all cohorts that received the agent.

In AGAVE-201, serious adverse reactions affected 44% of patients who received axatilimab-csfr. Adverse reactions led to dose interruption in 44% of patients, dose reduction in 8% of patients, and permanent discontinuation of the agent in 10% of patients. Reactions that affected 15% or more of patients were bacterial infection, cough, diarrhea, dyspnea, fatigue, headache, musculoskeletal pain, nausea, pyrexia, unspecified-pathogen infection, and viral infection, along with laboratory abnormalities. Eighteen percent of recipients had infusion-related reactions, and in 1.3%, these were grade 3 or 4 severity.

Axatilimab-csfr has also been added to the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology, with a category 2A recommendation to treat chronic GVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients who weigh at least 40 kg.

Clinical trials are proceeding to investigate the frontline use of axatilimab-csfr in combination with ruxolitinib for chronic GVHD, as well as with steroids. It is also being evaluated for treatment of idiopathic pulmonary fibrosis.

Reference

Incyte and Syndax announce U.S. Food and Drug Administration (FDA) approval of Niktimvo™ (axatilimab-csfr) 9 mg and 22 mg vial sizes. Press Release. PR Newswire. January 15, 2025. Accessed January 16, 2025.