From discovery to approval: a brief history of the compstatin family of complement C3 inhibitors
The FDA approval of pegcetacoplan (Empaveli), a PEGylated compstatin-based C3
therapeutic, as a new treatment for paroxysmal nocturnal hemoglobinuria (PNH) marks a
milestone in the history of complement drug discovery. Almost 15 years after the approval of
the first complement-specific drug for PNH, the anti-C5 antibody eculizumab, a novel class of
complement inhibitors with a distinct mechanism of action finally enters the clinic. This
landmark decision broadens the spectrum of available complement therapeutics, offering …
therapeutic, as a new treatment for paroxysmal nocturnal hemoglobinuria (PNH) marks a
milestone in the history of complement drug discovery. Almost 15 years after the approval of
the first complement-specific drug for PNH, the anti-C5 antibody eculizumab, a novel class of
complement inhibitors with a distinct mechanism of action finally enters the clinic. This
landmark decision broadens the spectrum of available complement therapeutics, offering …