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Apellis and Sobi Announce EU Approval of Aspaveli® (pegcetacoplan) for Treatment of PNH
The 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
About this update from Apellis Pharmaceuticals, Inc.
[{"type":"text","content":"The 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 hemoglobin levels1 Aspaveli will have market exclusivity for paroxysmal nocturnal hemoglobinuria (PNH) based on orphan drug designation WALTHAM, Mass. and STOCKHOLM, Sweden, Dec. 15, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) 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 hemoglobinuria (PNH) who are anemic 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. PNH 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 hemolysis and extravascular hemolysis. Characterized by persistently low hemoglobin, PNH can result in frequent transfusions and debilitating symptoms such as severe fatigue caused by anemia. Despite improvements in hemolytic activity with C5 inhibitor treatment, approximately 72% of people with PNH treated with C5 inhibitors remain anemic, according to a retrospective and a cross-sectional study.2,3 “The European Commission’s approval of Aspaveli is a milestone for people living with PNH across Europe,” said Guido Oelkers, chief executive officer 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.” “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, M.D., Ph.D., chief medical officer of Apellis. “Today's approval represents the first new class of complement medicines in Europe in over a decade,...