In August 2023, the U.S. FDA approved pozelimab-bbfg (Veopoz), developed by Regeneron Pharmaceuticals, as the first targeted therapy for CHAPLE (CD55-deficiency with hyperactivation of complement, angiopathic thrombosis, and protein-losing enteropathy). Approved for both adults and pediatric patients over 1 year of age, this milestone marks a major breakthrough in treating this ultra-rare complement-mediated disease. The approval underscores the potential of complement inhibition therapies in rare immunological disorders
