Introduction
Pegcetacoplan is the first medication approved for treating paroxysmal nocturnal hemoglobinuria (PNH). It binds to and inhibits the complement protein C3. Pegcetacoplan received approval for medical use in the United States in May 2021. The U.S. Food and Drug Administration (FDA) designates it as a first-in-class drug.

Mechanism of Action
Pegcetacoplan binds to complement protein C3 and its activation fragment C3b, thereby regulating the cleavage of C3 and the generation of downstream effectors of complement activation. Acting proximally in the complement cascade, it controls both C3b-mediated extravascular hemolysis and terminal complement-mediated intravascular hemolysis.

Indications
Treatment of paroxysmal nocturnal hemoglobinuria (PNH).