EU Approves Apellis and Sobi's Aspaveli for PNH with Restricted Label
Key Insights
The European Commission has approved pegcetacoplan (Aspaveli) for treating PNH (search) in adults with anemia, but only after three months of C5 (search) inhibitor therapy, unlike its broader US approval.
Clinical trials demonstrated Aspaveli's superiority over Soliris, with patients showing an average 2.4 g/dL increase in hemoglobin levels and 85% achieving transfusion independence over 16 weeks.
The approval marks a significant milestone in Apellis and Sobi (search)'s $1.25 billion partnership, with the drug showing potential for expansion into other complement-mediated diseases.
The European Commission has granted approval for pegcetacoplan (Aspaveli) in the treatment of paroxysmal nocturnal hemoglobinuria (search) (PNH (search)), marking a significant but restricted entry into the European market for Apellis Pharmaceuticals and Swedish Orphan Biovitrum (Sobi (search)). Unlike its US counterpart Empaveli, the European approval comes with a narrower scope, limiting its use to adults with PNH who remain anemic after at least three months of C5 (search) inhibitor treatment.
Clinical Efficacy and Treatment Advantages
The approval is supported by compelling data from the phase 3 PEGASUS study, which enrolled 80 PNH (search) patients with persistent anemia despite treatment with Soliris. The trial results demonstrated pegcetacoplan's superior efficacy, with treated patients experiencing an average hemoglobin increase of 2.4 g/dL, while the Soliris control group showed a decline of 1.5 g/dL. Notably, 85% of patients receiving pegcetacoplan remained transfusion-free over the 16-week study period, compared to only 15% in the Soliris group.
A key advantage of Aspaveli lies in its administration method - twice-weekly subcutaneous injections, offering a more convenient alternative to the intravenous infusions required for existing C5 (search) inhibitors like Soliris and Ultomiris.
Market Impact and Disease Context
PNH (search), a rare and life-threatening blood disorder, causes red blood cell destruction, blood clots, and impaired bone marrow function. Patients typically experience severe fatigue, recurring infections, and anemia often requiring regular transfusions. According to Apellis, approximately 75% of PNH patients remain anemic despite C5 (search) inhibitor treatment, highlighting a significant unmet need that Aspaveli could address.
Future Development Pipeline
The approval represents a crucial milestone in the $1.25 billion partnership between Apellis and Sobi (search), who share development responsibilities and ex-US marketing rights. The companies are actively expanding pegcetacoplan's potential applications, with ongoing development programs in:
- Geographic atrophy
- Immune complex membranoproliferative glomerulonephritis (IC-MPGN)
- C3 glomerulopathy (C3G)
- Cold agglutinin disease (CAD)
- Hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA)
- Amyotrophic lateral sclerosis (ALS)
Early market performance in the US has been promising, with Empaveli generating over $5 million in third-quarter sales, exceeding initial expectations. Industry analysts at Jefferies project the drug could potentially reach $4 billion in peak sales.