- • If approved, pozelimab would be the first and only treatment for those living with CHAPLE.
- • CHAPLE is an ultra-rare hereditary immune disease that causes overactivation of the complement system, leading to potentially life-threatening abdominal and cardiovascular symptoms.
Regeneron Pharmaceuticals, Inc. has announced the U.S. Food and Drug Administration has accepted for Priority Review the Biologics License Application (BLA) for pozelimab as a treatment for adults and children as young as 1 year of age with CHAPLE disease (also known as CD55 deficiency with Hyperactivation of complement, Angiopathic thrombosis and Protein Losing Enteropathy or CD55-deficient protein-losing enteropathy). There are currently no approved treatments for CHAPLE, an ultra-rare and life-threatening hereditary immune disease driven by an overactivation of the complement system. Pozelimab is an investigational fully-human monoclonal antibody designed to block the activity of complement factor C5, a protein involved in complement system activation. The target action date for the FDA decision is August 20, 2023.
