pegcetacoplan

Pegcetacoplan is a pegylated peptide complement inhibitor that selectively binds to complement component 3 (C3) and its activation fragment C3b, thereby inhibiting the cleavage of C3 into C3a and C3b. This action blocks all three complement activation pathways (classical, alternative, and lectin), preventing downstream effects such as opsonization, inflammation, and formation of the membrane attack complex (MAC). Pegcetacoplan is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adults via subcutaneous infusion and for geographic atrophy secondary to age-related macular degeneration via intravitreal injection. It was developed to address both intravascular hemolysis mediated by MAC formation and extravascular hemolysis mediated by C3b opsonization in PNH patients. Pegcetacoplan is considered first-in-class as a proximal complement inhibitor targeting C3.

Brand names

EmpaveliSyfovre

Other names

pegcetacoplan