BRIEF—Apellis and Sobi receive EU approval for Aspaveli

The European Commission has approved Aspaveli (pegcetacoplan), developed by Apellis and Swedish Orphan Biovitrum (also known as Sobi), the first and only 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.

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 C5 treated patients remain anemic, according to a retrospective and cross-sectional study.

Pegcetacoplan was also approved in May for the treatment of PNH in the U.S. under the brand name Empaveli.