Apellis Pharmaceuticals, Inc. and Swedish Orphan Biovitrum AB (publ) announced 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. 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. 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.