FDA Approves Axatilimab-csfr 9-mg, 22-mg Vial Use for Chronic GVHD

The FDA has issued approval of 9-mg and 22-mg vial axatilimab-csfr to treat chronic graft-versus-host disease (GVHD) after failure of at least two lines of systemic therapy. The approval is for use in adult and pediatric patients who weigh at least 40 kg, dosed 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, two global biopharmaceutical companies which co-develop and commercialize axatilimab-csfr as Niktimvo. Niktimvo is a trademark of Incyte and the agent is expected to be available 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,” stated Incyte Chief Executive Officer Hervé Hoppenot.

Axatilimab-csfr is an antibody which blocks the colony-stimulating factor-1 receptor (CSF-1R), treating chronic GVHD by 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,” commented Syndax CEO Michael Metzger.

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

Regarding safety findings 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. Specific reaction types 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, as well as laboratory abnormalities. There were infusion-related reactions in 18% of recipients and in 1.3% these were grade 3 or 4 severity.

Axatilimab-csfr is also now included in the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology. It has 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 currently testing frontline use of axatilimab-csfr in combination with ruxolitinib to treat 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.