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 haemoglobin levels 1.
| STOCKHOLM, Dec. 15, 2021 /PRNewswire/ --
Swedish 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. 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 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 “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.” “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 of this important treatment in the United States.” The approval is based on the results from the head-to-head PEGASUS phase 3 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 full safety and efficacy results were published in The New England Journal of Medicine in March 2021.1 Orphan drug designation is granted to therapies that treat a serious disease that affects fewer than five in 10,000 people in the EU and provide a significant benefit over existing treatments. Aspaveli will have market exclusivity based on orphan drug designation for PNH. About Aspaveli®/Empaveli™ About the Sobi and Apellis Collaboration About Apellis About Sobi™ Contacts Sobi To contact the Sobi Investor Relations Team, click here. For Sobi Media contacts, click here. Apellis Media: Investors: Reference 1. Hillmen P, Szer J, Weitz I, et al. Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. DOI: 10.1056/NEJMoa2029073. 2. McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471. 3. Dingli ASH 2020 Abstract/ p.1/ Methods/ ln.1-2; p.2/ Results/ln.7-9; ln.14-1. 4. Risitano et al. 2009. This information was brought to you by Cision http://news.cision.com The following files are available for download:
SOURCE Swedish Orphan Biovitrum AB | ||||
Company Codes: Bloomberg:SOBI@SS, ISIN:SE0000872095, RICS:SOBI.ST, Stockholm:SOBI |
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