Pegcetacoplan

BeschreibungPegcetacoplan: a pegylated complement C3 inhibitor peptide.Pegcetacoplan is a complement inhibitor indicated in treating paroxysmal nocturnal hemoglobinuria (PNH). Before its FDA approval, patients with PNH were typically treated with the C5-inhibiting monoclonal antibody eculizumab. Patients given eculizumab experienced less hemolysis caused by the membrane attack complex but were still somewhat susceptible to hemolysis caused by C3b opsonization. Pegcetacoplan was developed out of a need for an inhibitor of complement-mediated hemolysis further upstream of C5. Pegcetacoplan is a pegylated C3 inhibitor that can disrupt the processes, leading to both forms of hemolysis that threaten patients with PNH. Pegcetacoplan for subcutaneous use was granted FDA approval on 14 May 2021.7 In February 2023, the FDA approved pegcetacoplan for intravitreal use for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration.

HistoryPegcetacoplan is the first treatment for paroxysmal nocturnal hemoglobinuria that binds to and inhibits complement protein C3. Its injection form names syfovre. Pegcetacoplan was approved for medical use in the United States in May 2021. The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.In 2024, the American Society of Nephrology Annual Kidney Meeting, the study group who investigating pegcetacoplan in the largest multicenter double-blind VALIANT trial, showed its significant benefits in the treatment of patients with C3 glomerulopathy or primary immune complex–mediated membranoproliferative glomerulonephritis.