Pegcetacoplan: A New Opportunity for Complement Inhibition in PNH

J Blood Med. 2023 Mar 29:14:239-245. doi: 10.2147/JBM.S362220. eCollection 2023.


Pegcetacoplan is the newest inhibitor of the complement system to be approved by the FDA and EMA for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). The cyclic peptide inhibitor of C3 was evaluated in several clinical trials in PHN leading to its approval. The focus of this paper will review the efficacy and safety of Pegcetacoplan (PEG), and considerations for use in patients with PNH.

Keywords: PNH; complement inhibition; extravascular hemolysis.

Publication types

  • Review

Grants and funding

No honoraria were paid for this review.