New Immune-suppressive Therapy for Graft-versus-host Disease
The FDA approved axatilimab-csfr, an inhibitor of monocytes and macrophages, for certain patients with chronic graft-versus-host disease.
The U.S. Food and Drug Administration (FDA) has approved axatilimab-csfr (Niktimvo) for the treatment of adult and pediatric patients weighing at least 40 kg with chronic graft-versus-host disease (cGVHD) who have not responded to at least two prior lines of systemic therapy.
Axatilimab-csfr is a targeted therapy that blocks the protein CSF-1R on the surface of immune cells called monocytes and macrophages. During cGVHD, a side effect that can arise from stem cell transplants for blood cancers, monocytes and macrophages contribute to the widespread inflammation that can damage a patient’s healthy tissues. By blocking CSF-1R, axatilimab-csfr impairs the function of monocytes and macrophages to help control the disease.
This is the first FDA approval for axatilimab-csfr.
The approval was based on results from the randomized, open-label, multicenter phase II AGAVE-201 clinical trial that tested three dose levels of axatilimab-csfr in adult and pediatric patients with cGVHD who needed additional treatment after two or more lines of prior systemic therapy.
Among 79 patients treated with the recommended dose, 75% had experienced a response in the first 24 weeks of treatment, and the responses endured for a median of 1.9 months. Sixty percent of those who had a response survived without requiring additional systemic therapies for at least 12 months.
The recommended dose of axatilimab-csfr is 0.3 mg/kg to a maximum dose of 35 mg. The drug is delivered as a 30-minute infusion every two weeks until disease progression or unacceptable toxicity.
cGVHD is a condition in which donor immune cells from a stem cell transplant recognize the recipient’s body as foreign and attack healthy tissues. It differs from acute GVHD in that it is often diagnosed later (100 or more days post-transplant). It is estimated that over 40% of stem cell transplant recipients may develop cGVHD up to three years post-treatment.
The FDA rendered its decision on August 14, 2024.
