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Aspaveli® (pegcetacoplan) approved in EU as orphan drug for treatment of PNH
STOCKHOLM, Dec. 15, 2021 /PRNewswire/ -- The first targeted C3 therapy approved in the EU Approval based on results from head-to-head PEGASUS phase 3 study
About this update from Apellis Pharmaceuticals, Inc.
[{"type":"text","content":"STOCKHOLM, Dec. 15, 2021 /PRNewswire/ --\nThe first targeted C3 therapy approved in the EU Approval based on results from head-to-head PEGASUS phase 3 study where Aspaveli demonstrated superiority to eculizumab in improving haemoglobin levels1 Aspaveli will have market exclusivity for paroxysmal nocturnal haemoglobinuria (PNH) based on orphan drug designationSwedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) and Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) announced today that the European Commission (EC) has approved Aspaveli® (pegcetacoplan), the first and only targeted C3 therapy, for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least three months. Based on the recommendation from the European Medicines Agency's Committee for Orphan Medicinal Products, the EC determined that pegcetacoplan continues to meet the criteria for the orphan drug designation status granted in 2017 for the treatment of PNH.\nPNH is a rare, chronic and life-threatening blood disorder where uncontrolled complement activation leads to the destruction of oxygen-carrying red blood cells through intravascular haemolysis and extravascular haemolysis. Characterised by persistently low haemoglobin, PNH can result in frequent transfusions and debilitating symptoms such as severe fatigue caused by anaemia. Despite improvements in haemolytic activity with C5 inhibitor treatment, approximately 72 per cent of people with PNH treated with C5 inhibitors remain anaemic, according to a retrospective and a cross-sectional study.2,3 \n\"The European Commission's approval of Aspaveli is a milestone for people living with PNH across Europe,\" said,\" Guido Oelkers, CEO and President at Sobi. \"The symptoms of PNH can significantly impact quality of life. In addition, despite current therapy, many people still require frequent blood transfusions. We are now working with EU member states to provide access to this important medicine as quickly as possible.\"\n\"As the first and only targeted C3 therapy in Europe, Aspaveli has the potential to elevate the standard of care for patients living with PNH,\" said Federico Grossi, MD, PhD, Chief Medical Officer of Apellis. \"Today's approval represents the first new class of complement medicines in Europe in over a decade, building on the launch...