Corey Cutler, MD, Director of Adult Stem Cell Transplantation Program at Dana-Farber Cancer Institute in Boston and a Professor of Medicine at Harvard Medical School, discusses the approval of Niktimvo (axatilimab) for the treatment of chronic graft versus host disease (GVHD).
Chronic GVHD is a rare complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted donor cells attack the transplant recipient’s body. Symptoms may include skin rash, mouth sores, dry eyes, liver inflammation, development of scar tissue in the skin and joints, and damage to the lungs. The exact cause of chronic GVHD is unknown. It likely results from a complex immune-mediated interaction between the donor and recipient cells.
The U.S. Food and Drug Administration (FDA) has approved axatilimab for the treatment of patients with chronic GVHD after failure of two or more lines of systemic therapy for adult and pediatric patients weighing at least 40 kg. Axatilimab is the first anti-CSF-1R antibody for this indication.
This approval follows results from the AGAVE-201 clinical trial, a phase 2, open-label, randomized, multicenter study testing the efficacy, safety, and tolerability of axatilimab in patients with refractory chronic GVHD. The trial also consisted of dose ranging where patients were randomized to one of three groups with a distinct dose. The primary endpoint was the proportion of patients in each dose group who achieved an objective response as defined by the 2014 NIH Consensus Criteria for chronic GVHD by cycle 7 day 1.
The most effective dose was 0.3 mg/kg every two weeks which had the highest overall response rate and least toxicity. Additionally, durable responses in all organs and patient subgroups were observed.
To learn more about chronic graft versus host disease and other rare autoimmune disorders, visit https://checkrare.com/diseases/autoimmune-auto-inflammatory-disorders/
