RARE Daily

FDA Grants Priority Review for Incyte’s Axatilimab for Treatment of GVHD

February 28, 2024

Rare Daily Staff

The U.S. Food and Drug Administration has accepted for Priority Review Incyte’s Biologics License Application for axatilimab, an anti-CSF-1R antibody, for the treatment of chronic graft-versus-host disease 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.

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 percent of transplant recipients and affects approximately 14,000 patients in the United States. Chronic GVHD typically manifests across multiple organ systems, with skin and mucosa being commonly involved, and is characterized by the development of fibrotic tissue.

Axatilimab is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages along with their monocyte precursors, which has been shown to play a key role in the fibrotic disease process underlying diseases such as chronic GVHD and idiopathic pulmonary fibrosis.

The BLA is supported by positive data from the AGAVE-201 trial, 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.

“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, CEO of Incyte. “Axatilimab’s novel mechanism offers a differentiated treatment approach which may help patients suffering from this devastating disease.”

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.

In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab. Axatilimab is being developed under an exclusive worldwide license from UCB entered into between Syndax and UCB in 2016.

Photo: Hervé Hoppenot, CEO of Incyte

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