Pegcetacoplan

Generic Name: Pegcetacoplan

Brand Names: Empaveli, Syfovre, Aspaveli

Drug Type: Biotech

Chemical Formula: -

CAS Number: 2019171-69-6

Unique Ingredient Identifier: TO3JYR3BOU

Background: Pegcetacoplan is a complement inhibitor indicated in the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Prior to 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. In February 2023, pegcetacoplan for intravitreal use was approved by the FDA for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration.

Indication: Pegcetacoplan is indicated to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). It is also indicated to treat geographic atrophy (GA) secondary to age-related macular degeneration.

Associated Conditions: Geographic Atrophy Secondary to Age-related Macular Degeneration, Paroxysmal Nocturnal Haemoglobinuria (PNH)

Associated Therapies: -

Clinical Trials

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