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FDA Sets Action Date for Axatilimab in Graft-Versus-Host Disease


The Prescription Drug User Fee Act action date is Aug. 28, 2024.

The FDA has accepted for priority review Incyte’s biologics license application (BLA) for axatilimab to treat patients with 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 is Aug. 28, 2024.

Graft-versus-host disease is a complication of stem cell transplants, where the body’s immune system attacks the new cells. Acute graft-versus-host can occur in about half of patients receiving hematopoietic stem cell transplantation from matched donors. The occurrence can be higher in unmatched donors

Axatilimab, developed by Incyte and Syndax Pharmaceuticals, is a monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein.

The BLA is supported by 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. This phase 1/2 study enrolled 40 patients who received at least one dose of axatilimab. At least one treatment-related adverse event (TRAE) was observed in 30 patients. The overall response rate in the first six cycles, an endpoint for regulatory review, was 82%. Additionally, 58% of patients reported improvement in symptoms.

Data were published in the Journal of Clinical Oncology. Additional data were presented at the American Society of Hematology Annual Meeting 2023. The trial met the primary endpoint across all cohorts receiving axatilimab. Patients who received axatilimab at 0.3 mg/kg every two weeks achieved the highest overall response rate of 74% within the first six months of treatment. This is the recommended dose of axatilimab that will be used for future trials in chronic graft-versus-host-disease.

In the overall trial population, 33% of patients experienced at least one grade ≥3 treatment-emergent adverse event, with 15.5% experiencing adverse events leading to discontinuation of treatment. For the 79 patients who received axatilimab at 0.3 mg/kg grade ≥3 treatment-emergent adverse events occurred in 17.7% of patients, with 6.3% leading to discontinuation of treatment.

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