Incyte Announces U.S. Food and Drug Administration Grants Priority Review for Axatilimab for the Treatment of Chronic Graft-Versus-Host Disease

Incyte (Nasdaq:INCY) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for axatilimab, an anti-CSF-1R antibody, for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy. The Prescription Drug User Fee Act (PDUFA) date for the FDA decision is August 28, 2024.

The BLA is supported by positive data from the AGAVE-201 trial (NCT04710576), recently highlighted in a Plenary Scientific Session at the American Society of Hematology Annual Meeting 2023, which showed that treatment with axatilimab resulted in clinically meaningful results and was generally well-tolerated, with a safety profile that was manageable and consistent with the mechanism of action of CSF-1R inhibition.

Axatilimab is being developed by Incyte and Syndax Pharmaceuticals (Nasdaq:SNDX) as part of an exclusive worldwide co-development and co-commercialization license agreement.

“Despite recent advancements in the treatment of patients with chronic GVHD, there remains a significant unmet need for patients who progressed on earlier lines of therapy,” said Hervé Hoppenot, Chief Executive Officer, Incyte. “Axatilimab’s novel mechanism offers a differentiated treatment approach which may help patients suffering from this devastating disease. We look forward to working closely with the FDA and our partners at Syndax on the review of our application for axatilimab for this indication.”

The FDA grants Priority Review designation to applications for medicines that, if approved, would treat a serious condition and provide significant improvements in the safety or effectiveness of the treatment.

About Chronic Graft-Versus-Host Disease

Chronic graft-versus-host disease (GVHD), an immune response of the donor-derived hematopoietic cells against recipient tissues, is a serious, potentially life-threatening complication of allogeneic hematopoietic stem cell transplantation which can last for years. Chronic GVHD is estimated to develop in approximately 40% of transplant recipients, and affects approximately 14,000 patients in the U.S.^1,2. Chronic GVHD typically manifests across multiple organ systems, with skin and mucosa being commonly involved, and is characterized by the development of fibrotic tissue³.