Bone Marrow Failure Disorders

In a small, open-label phase Ib multicenter study, the PEGylated C3 inhibitor pegcetacoplan was found to potentially provide more complete hemolysis control in patients with paroxysmal nocturnal hemoglobinuria (PNH). It was well tolerated overall and yielded an improved hematological response due to broad hemolysis control through inhibition of C3, the investigators wrote. In conclusion, they said the study “demonstrated for the first time (to our knowledge) that systematic C3 inhibition using pegcetacoplan can be achieved safely and effectively for at least 2 years and enabled eculizumab discontinuation in four subjects.”