Empaveli (pegcetacoplan) moved complement inhibition upstream to C3, addressing the extravascular hemolysis that leaves many paroxysmal nocturnal hemoglobinuria patients anemic despite C5 blockade.
By blocking complement at C3 rather than C5, Empaveli controls red cell destruction both inside and outside the blood vessels, addressing the extravascular hemolysis that leaves many patients anemic. It is delivered as a twice-weekly subcutaneous infusion via a small pump for adults with paroxysmal nocturnal hemoglobinuria.
Empaveli faces no biosimilar competition, and pegcetacoplan has since been approved in ophthalmology for geographic atrophy, broadening the molecule’s value considerably. Its upstream mechanism and self-administered format make it the key comparator for newer oral and subcutaneous complement inhibitors.
