Sobi Receives Positive CHMP Opinion for Aspaveli® (pegcetacoplan) for the Treatment of C3G and Primary IC-MPGN

The CHMP has issued a positive opinion recommending EU marketing authorisation for Aspaveli (pegcetacoplan) to treat adults and adolescents (12+) with C3 glomerulopathy (C3G) and primary immune-complex MPGN, with the decision now pending at the European Commission and expected in Q1 2026; the recommendation is based on Phase 3 VALIANT data (n=124) showing significant reductions in proteinuria, stabilization of kidney function and clearance of C3 deposits. If approved, Aspaveli would be the first approved therapy in Europe for these rare kidney diseases—affecting roughly 8,000 patients in Europe and associated with >50% progression to kidney failure within 5–10 years—creating a high-unmet-need orphan market. Commercially, Sobi (exclusive ex-U.S. rights) and partner Apellis (U.S. and ophthalmology rights) would gain a new specialty indication that could meaningfully augment Sobi’s rare-disease franchise (SEK 26bn revenue in 2024), though ultimate financial impact will depend on final approval, pricing and reimbursement dynamics.

Analysis

The CHMP has issued a positive opinion recommending marketing authorisation for Aspaveli (pegcetacoplan) to treat adults and adolescents (12+) with C3 glomerulopathy (C3G) and primary IC-MPGN; the recommendation is now with the European Commission, which is expected to decide in Q1 2026. These diseases affect roughly 8,000 patients in Europe and have high unmet need, with more than 50% of patients progressing to kidney failure within five to ten years, creating a clear orphan-market opportunity if approved.

The CHMP recommendation is supported by the Phase 3 VALIANT trial (n=124), which met its primary endpoint (log-transformed UPCR at Week 26) and showed significant reductions in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits; these results were published in The New England Journal of Medicine. VALIANT is the largest randomized trial in these populations and included adolescents and both native and post-transplant kidneys, strengthening the regulatory case for a labelled indication in patients 12 and older.

Commercially, Sobi holds exclusive ex-U.S. commercialization rights while Apellis retains U.S. rights and ophthalmology indications; Sobi reported SEK 26 billion revenue in 2024. Sentiment and market-impact signals are moderately positive overall (sentiment_score 0.5; SOBI 0.7, APLS 0.4), but ultimate value depends on the European Commission ruling, subsequent pricing/reimbursement outcomes, and real-world uptake and safety data post-approval.

Key Decisions for Investors

• Consider increasing selective exposure to SOBI ahead of the European Commission decision expected in Q1 2026 while sizing positions to reflect reimbursement and uptake uncertainty
• Monitor the EC decision, subsequent national reimbursement decisions and early real-world uptake (prescriptions, coverage) and be prepared to trim or add exposure based on pricing outcomes and roll-out evidence
• Maintain a cautious or modest position in Apellis pending U.S. commercial execution and broader ophthalmology franchise performance given lower sentiment and more contingent upside
• Use event-sized hedges (options or position limits) to protect against downside from regulatory delays, adverse post-marketing safety signals or unfavorable reimbursement outcomes