Pegcetacoplan: the first and only C3-targeted therapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria.

Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disorder of hematopoietic stem cells, characterized by somatic mutations of the Phosphatidylinositol Glycan Class A Gene, resulting in increased hemolysis. The advent of complement inhibitors has since changed the way clinicians approach treating PNH. Pegcetacoplan is a C3 inhibitor that has shown promise in this field and improved outcomes for patients who have been diagnosed with PNH.

Areas covered: This review article will aim to examine the pathophysiology of PNH and the current treatments available, with a focus on pegcetacoplan. It will focus on the pharmacodynamics, pharmocokinetics and evidence in the use of pegcetacoplan in PNH. Electronic sources including PubMed, MEDLINE, were utilized with studies in the last 5 years prioritized, especially the phase 3 Prince and Pegasus studies.

Expert opinion: The results from phase 3 studies for pegcetacoplan have been promising, showing good efficacy and improvements in patients' conditions. More research is required to evaluate the use of pegcetacoplan, especially in combination with existing treatment in patients who are having suboptimal results. Nonetheless, with more results on the way and new agents to treat PNH in the vicinity, this remains a very exciting time for both clinicians and patients.