Pegcetacoplan

C1970H3848N50O947S4
Pegcetacoplan is a complement inhibitor approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Before it received FDA authorization, the standard therapy for PNH patients was eculizumab, a monoclonal antibody that targets C5. While eculizumab effectively reduced membrane attack complex-induced hemolysis, it did not prevent hemolysis mediated by C3b opsonization, leaving patients still vulnerable to this form of red blood cell destruction. This unmet need led to the development of pegcetacoplan, a complement inhibitor that acts upstream of C5 to block complement-mediated hemolysis at an earlier stage. As a pegylated C3 inhibitor, pegcetacoplan can inhibit the pathological processes underlying both types of hemolysis that affect PNH patients.

Pegcetacoplan for subcutaneous administration was approved by the FDA on May 14, 2021. In February 2023, the FDA further approved intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) associated with age-related macular degeneration.

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