Safety and efficacy of pegcetacoplan in paroxysmal nocturnal hemoglobinuria.

86 , 87 In addition, although pegcetacoplan-treated patients with GA secondary to AMD in the phase III DERBY trial ( NCT03525600 ) narrowly missed the primary endpoint of reduction in GA lesions at Month 12 of the study, 86 longer-term analysis of patients has demonstrated that pegcetacoplan contributed to nominally significant reductions in GA lesion growth at Month 18. 87 Pegcetacoplan was also associated with a 73.3% reduction in proteinuria in five patients with C3 glomerulopathy in the phase II DISCOVERY trial ( NCT03453619 ) 88 and demonstrated preliminary efficacy in patients with cold agglutinin disease in the PLAUDIT trial ( NCT03226678 ). 89 It is likely that pegcetacoplan’s approved usage will be expanded to some of these conditions in the future, although the dosage and route of administration remains to be determined.

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