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The major clinical complications of paroxysmal nocturnal hemoglobinuria (PNH) include a pronounced symptom burden (fatigue, abdominal pain, chest pain), anemia (as well as other cytopenias), and increased thrombosis risk. Terminal complement C5 protein inhibition with eculizumab improves symptoms, ameliorates anemia by reducing intravascular hemolysis, and reduces thrombosis risk. However, anemia may not fully resolve, as PNH red cells opsonized with complement C3 protein are still susceptible to extravascular hemolysis.
Pegcetacoplan, which targets C3, was developed with the aim of addressing both extravascular and intravascular hemolysis by proximal inhibition of the complement pathway. Investigators conducted an industry-funded randomized…