Pegcetacoplan binds to complement protein C3 and C3b, preventing the activation of the complement cascade. This targeted inhibition effectively reduces:
Reduces hemolysis and inflammation in paroxysmal nocturnal hemoglobinuria (PNH).
Prevents retinal cell damage associated with geographic atrophy in age-related macular degeneration.
Mitigates immune-mediated tissue injury in other complement-driven disorders.
Pegcetacoplan API is an active pharmaceutical ingredient consisting of a pegylated cyclic peptide designed to regulate the immune system by acting as a targeted C3 complement inhibitor.
It binds specifically to complement proteins C3 and C3b, effectively preventing the downstream activation of the complement cascade that causes cell damage and inflammation.
It is primarily developed for complement-mediated diseases, including Paroxysmal Nocturnal Hemoglobinuria (PNH) and Geographic Atrophy (GA) secondary to age-related macular degeneration.
By controlling C3 complement activation, Pegcetacoplan reduces both intravascular and extravascular hemolysis as well as systemic inflammation in patients with PNH.
It slows the progression of geographic atrophy by protecting retinal cells from complement-driven tissue destruction and degeneration.