HematologyFDA Approved

Pegcetacoplan (Empaveli)

Subcutaneous infusion

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).

Mechanism of Action

This dual mechanism addresses the limitation of C5 inhibitors that only prevent intravascular hemolysis..

Research Summary & Key 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.

Clinical Status

FDA Approved

Pegcetacoplan (Empaveli) has received FDA approval and is available for clinical use under appropriate medical supervision. Consult a qualified healthcare provider for prescribing information.

Administration Routes

Subcutaneous infusion

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Disclaimer: This information is provided for educational and research purposes only. It is not intended as medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before starting any peptide therapy. The Peptide Association does not endorse or recommend any specific treatment protocol.