Aspaveli® (pegcetacoplan) receives positive CHMP opinion for treatment of PNH

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[{"type":"text","content":"STOCKHOLM, Oct. 15, 2021 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) and Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) announce today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion recommending the marketing authorisation of Aspaveli® (pegcetacoplan) for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least three months. The positive opinion from the CHMP is now referred to the European Commission for an approval decision.\nPNH is a rare, chronic, life-threatening blood disorder where uncontrolled complement activation leads to the destruction of oxygen-carrying red blood cells through intravascular and extravascular haemolysis. Characterised by persistently low haemoglobin, PNH can result in frequent transfusions and debilitating symptoms such as severe fatigue. Despite improvements in haemolytic activity with C5 inhibitor treatment, approximately 72 per cent of C5-treated patients remain anaemic, according to a retrospective and a cross-sectional study.1,2\n\"Today's positive opinion by the CHMP is a significant milestone for people living with PNH across Europe,\" said Ravi Rao, Head of Research & Development and Chief Medical Officer at Sobi. \"We hope to make a difference to the lives of people living with rare diseases, and if Aspaveli is approved by the European Commission, it will offer patients and treating physicians a new class of complement medicines for the treatment of PNH.\"\nThe positive opinion is based on the results from the head-to-head phase 3 PEGASUS study, which evaluated the efficacy and safety of Aspaveli compared to eculizumab at 16 weeks in adults with PNH who had persistent anaemia despite treatment with eculizumab. The results were published in The New England Journal of Medicine in March 2021.2\n\"Building on our recent U.S. approval, the positive CHMP opinion moves us one step closer towards bringing this important treatment to patients across Europe,\" said Federico Grossi, MD, PhD, Chief Medical Officer at Apellis. \"If approved, Aspaveli has the potential to redefine treatment for patients with PNH, so we look forward to the European Commission's final decision.\"\nAspaveli is the European trade name for pegcet...

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