Apellis reports positive top-line results from phase III PEGASUS head-to-head study of pegcetacoplan compared to eculizumab for paroxysmal nocturnal hemoglobinuria.
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Alexion Pharmaceuticals, Inc. announced the FDA has approved the expanded use of Ultomiris (ravulizumab-cwvz) to include children (one month of age and older) and adolescents with paroxysmal nocturnal hemoglobinuria (PNH). Ultomiris, a long-acting C5 inhibitor that offers immediate, complete and sustained complement inhibition, is now the first and only FDA-approved medicine for children and adolescents with PNH.
Swedish Orphan Biovitrum AB (publ) (Sobi™) and Apellis Pharmaceuticals, Inc. announced that The New England Journal of Medicine (NEJM) published results from the phase III PEGASUS study, which demonstrated the superiority of pegcetacoplan, an investigational targeted C3 therapy, in improving haemoglobin levels and showed improvements in key clinical outcomes compared to eculizumab, a C5 inhibitor, in adults with paroxysmal nocturnal haemoglobinuria (PNH) at 16 weeks who had persistent anaemia following treatment with eculizumab.