compstatin

Compstatin is a cyclic tridecapeptide that acts as a potent and selective inhibitor of the complement system by binding to complement component 3 (C3) and its activation fragment C3b. By binding to C3, compstatin sterically hinders the access of C3 convertases to their substrate, thereby preventing the cleavage of C3 into C3a and C3b. This inhibition effectively blocks all three pathways of complement activation (classical, lectin, and alternative). Originally discovered through phage-display library screening at the University of Pennsylvania, compstatin has served as the scaffold for several clinical-stage analogs, including pegcetacoplan (Empaveli) and AMY-101, developed for conditions such as paroxysmal nocturnal hemoglobinuria (PNH) and age-related macular degeneration (AMD).

Other names

cyclic tridecapeptide C3 inhibitor