Axatilimab-csfr received approval from the U.S. Food and Drug Administration (FDA) to treat chronic graft-versus-host disease (GVHD) in adult and pediatric (who weigh at least 88.2 pounds) patients who have received at least two prior lines of therapy. The treatment is an anti-CSF-1R antibody.
Approval was granted based on the results of the global AGAVE-201 study, which demonstrated a 75% overall response rate within the first six months of treatment. The median time to response was 1.5 months, and 60% of patients maintained a response at one year.
Serious adverse reactions occurred in 44% of patients and included bacterial infection, pyrexia, respiratory failure, viral infection, musculoskeletal pain, and infection (pathogen unspecified).
Common adverse reactions include musculoskeletal pain, pyrexia, increased alkaline phosphatase, increased amylase, fatigue, increased lipase, bacterial infection, infection (pathogen unspecified), increased aspartate aminotransferase, nausea, increased alanine aminotransferase, diarrhea, increased calcium, headache, cough, increased creatine phosphokinase, increased gamma glutamyl transferase, decreased phosphate, decreased hemoglobin, dyspnea, and viral infection.
“I am excited that [axatilimab-csfr] is designed to specifically target key drivers of inflammation and fibrosis in chronic GVHD, and I am highly encouraged by the robust responses observed across all organs and patient subgroups within the heavily pretreated population enrolled in the AGAVE-201 trial,” said Daniel Wolff, MD, PhD, head of the GVHD Center at University Hospital Regensburg in Germany. “I look forward to having a new and differentiated treatment option for my patients who need additional therapies to address this very difficult-to-manage, debilitating disease.”
Source: Business Wire, August 14, 2024.
