Pharma Focus Asia

FDA Accepts Priority Review for Pozelimab (C5 Antibody) BLA for Treatment of Children and Adults

Tuesday, February 21, 2023

Regeneron Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics Licence 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.

If approved, pozelimab will be the first and only treatment for people 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.

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