Pegcetacoplan (Empaveli)
Overview
A pegylated cyclic peptide that inhibits complement component C3, the central node of all three complement activation pathways (classical, lectin, and alternative). By binding C3 and preventing its cleavage into C3a and C3b, pegcetacoplan blocks both intravascular hemolysis (mediated by membrane attack complex) and extravascular hemolysis (mediated by C3b opsonization and phagocytosis). This dual mechanism addresses the limitation of C5 inhibitors that only prevent intravascular hemolysis.
Key Research Findings
FDA-approved in 2021 for paroxysmal nocturnal hemoglobinuria (PNH) in adults. PEGASUS trial demonstrated superior hemoglobin improvement over eculizumab (anti-C5), with adjusted hemoglobin difference of 3.84 g/dL (Hillmen et al., NEJM, 2021). Also FDA-approved for C3 glomerulopathy (2024). Subcutaneous self-administration via infusion pump allows home treatment.
Subcutaneous infusion
FDA Approved
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