The U.S. Food and Drug Administration (FDA) has approved Revuforj (axatilimab), a novel therapy developed by Syndax Pharmaceuticals, for the treatment of adult and pediatric patients aged 12 years and older with relapsed or refractory chronic graft-versus-host disease (cGVHD) after failure of one or two prior lines of systemic therapy. This approval marks a significant advancement in the treatment of cGVHD, providing a new option for patients with limited alternatives.
Mechanism of Action
Axatilimab is a humanized IgG1 antibody that targets colony stimulating factor 1 receptor (CSF-1R). CSF-1R is a key regulator of monocyte and macrophage function, and its inhibition by axatilimab is believed to modulate the immune response in cGVHD. The drug is administered intravenously.
Clinical Trial Data
The approval was based on data from the AGAVE-201 trial, a single-arm, open-label, multi-center study that evaluated the efficacy and safety of axatilimab in patients with relapsed or refractory cGVHD. The study included patients who had failed one or two prior lines of systemic therapy. The primary endpoint was overall response rate (ORR). Key secondary endpoints included duration of response (DOR) and changes in symptom burden.
The AGAVE-201 trial demonstrated a clinically meaningful ORR in patients treated with axatilimab. Detailed results from the trial have been presented at medical conferences and published in peer-reviewed journals, further supporting the efficacy and safety of axatilimab in this patient population.
cGVHD Disease Burden and Unmet Need
Graft-versus-host disease (GVHD) is a common complication following allogeneic hematopoietic stem cell transplantation (HSCT). Chronic GVHD (cGVHD) is a long-term condition that can affect multiple organ systems and significantly impact patients' quality of life. Despite advances in transplantation techniques and supportive care, cGVHD remains a major cause of morbidity and mortality in HSCT recipients. There remains a significant unmet need for effective therapies for patients with relapsed or refractory cGVHD who have failed prior lines of treatment.
Dosing and Administration
Revuforj (axatilimab) is administered intravenously. The recommended dose and schedule are available in the prescribing information. Healthcare professionals should consult the full prescribing information for detailed instructions on dosing, administration, and monitoring.
