ASPAVELI Fuels Sobi’s Growth Strategy in Europe for C3G and IC-MPGN

Summary 

• Sobi has received European Commission approval for ASPAVELI (pegcetacoplan) to treat C3G and primary IC-MPGN, two rare, progressive kidney diseases.
• The approval follows a positive CHMP opinion (December 2025) and marks the first approved treatment for primary IC-MPGN and the first for both C3G and IC-MPGN across adolescents and adults (aged ≥12 years).
• Approval is supported by data from the Phase 3 VALIANT trial (NCT05067127), the largest and first global study assessing efficacy and safety across pediatric, adult, and post-transplant groups.

Swedish Orphan Biovitrum (Sobi) has achieved a significant regulatory milestone with the European Commission’s approval of ASPAVELI (pegcetacoplan) for the treatment of two devastating rare kidney diseases. This decision marks a transformative moment for patients living with C3 glomerulopathy (C3G) and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN), conditions that have historically offered limited therapeutic options and posed severe risks of progression to end-stage kidney disease.

The European Commission approval represents far more than a regulatory clearance; it represents hope for an estimated 8,000 patients across Europe living with these progressive, life-altering kidney diseases. ASPAVELI is the first and only approved treatment for primary IC-MPGN and the first therapy indicated for both C3G and primary IC-MPGN across all patient age groups, including adolescents aged 12 to 17 years.

ASPAVELI will be administered in combination with a renin-angiotensin system (RAS) inhibitor, unless such treatment is contraindicated or poorly tolerated by the patient. This approval follows a positive Committee for Medicinal Products for Human Use (CHMP) opinion issued in December 2025, which reinforces the strength of the evidence supporting this innovative therapeutic approach.

C3G and primary IC-MPGN are rare, progressive kidney diseases characterized by dysregulation of the complement cascade, a critical component of the immune system. In healthy individuals, the complement system functions as a controlled defense mechanism; however, in patients with these conditions, excessive complement activation leads to the pathological accumulation of C3 deposits in the kidneys. This complement-driven inflammation triggers progressive kidney damage, proteinuria (protein loss in urine), and eventual renal failure.

​The clinical burden of these diseases is substantial. Approximately 50% of patients with C3G or primary IC-MPGN progress to end-stage kidney disease within five to ten years of diagnosis, requiring either kidney transplantation or dialysis, both of which carry significant morbidity and impact quality of life. Furthermore, for the subset of patients who receive transplants, disease recurrence occurs in approximately 90% of cases, underscoring the need for disease-modifying therapies that target the underlying pathophysiology rather than merely managing symptoms.

ASPAVELI is a targeted C3/C3b inhibitor designed to selectively regulate excessive complement activation at the C3 checkpoint of the complement cascade. By blocking this key step in complement activation, ASPAVELI prevents the formation of C3 convertases and the downstream amplification of the inflammatory cascade, thereby addressing the fundamental driver of kidney disease in C3G and primary IC-MPGN patients.

This mechanism-driven approach represents a paradigm shift in the therapeutic management of complement-mediated glomerulonephritis, moving away from non-specific immunosuppression toward precise molecular targeting of disease pathophysiology.

The European Commission approval is grounded in robust clinical evidence from the Phase 3 VALIANT study (NCT05067127), a landmark randomized, placebo-controlled, double-blind, multicenter trial that enrolled 124 patients aged 12 years and older with C3G or primary IC-MPGN. Notably, the VALIANT study is the largest single trial conducted in these patient populations and the first to simultaneously evaluate efficacy and safety across pediatric, adult, and post-transplant populations.

​Study participants were randomized to receive ASPAVELI or a placebo twice weekly for 26 weeks in the controlled phase, followed by a 26-week open-label extension in which all patients received active treatment. The primary endpoint assessed the log-transformed urine protein-to-creatinine ratio (UPCR) at Week 26 relative to baseline, a surrogate marker strongly associated with long-term renal protection and progression to end-stage kidney disease.

ASPAVELI demonstrated statistically significant and clinically meaningful benefits across all three major disease activity markers:

• Significant reduction in proteinuria – reflecting improved glomerular filtration barrier function
• Stabilization of glomerular filtration rate (eGFR) – indicating preservation of residual kidney function
• Substantial clearance of C3 deposits – demonstrating effective modulation of complement-driven inflammation

These results have been published in The New England Journal of Medicine, one of the most prestigious peer-reviewed medical journals, lending substantial credibility to ASPAVELI’s efficacy profile.

The approval of ASPAVELI addresses a critical unmet medical need. Until now, treatment options for C3G and primary IC-MPGN have been limited to non-specific immunosuppressive agents, many of which pose significant tolerability challenges and do not directly address the underlying complement dysregulation. The availability of a targeted, mechanism-based therapy has the potential to alter the natural history of these diseases, delaying or preventing progression to end-stage kidney disease and substantially improving quality of life for affected patients.

​According to Lydia Abad-Franch, MD, Head of R&D and Medical Affairs and Chief Medical Officer at Sobi, “The European Commission approval for ASPAVELI represents an important milestone for people living with C3G or primary IC-MPGN in Europe, two severe and rare kidney diseases with limited treatment options and a high risk of progression to kidney failure. This approval is underpinned by robust clinical evidence from the VALIANT trial, which demonstrated that pegcetacoplan is able to preserve kidney function by reducing proteinuria, stabilizing eGFR and clearing C3 deposits. ASPAVELI is now available for patients 12 years and older with these serious conditions.”

Sobi and Apellis Pharmaceuticals maintain a global co-development partnership for systemic pegcetacoplan. Under this collaboration, Sobi holds exclusive ex-U.S. commercialization rights for systemic pegcetacoplan across Europe and international markets, while Apellis retains U.S. commercialization rights and holds worldwide rights for ophthalmological applications of the therapy, including for geographic atrophy. Apellis previously obtained FDA approval for ASPAVELI in the United States for both C3G and primary IC-MPGN indications, making this a truly global therapeutic breakthrough.

This coordinated regulatory success across major markets, first in the United States and now in the European Union, positions ASPAVELI as the standard of care for patients with these rare but severe kidney diseases worldwide.In conclusion, the European Commission’s approval of ASPAVELI represents a transformative regulatory achievement in the treatment of rare kidney disease. By providing the first approved therapy for primary IC-MPGN and the first treatment for both C3G and IC-MPGN in adolescents and adults, ASPAVELI offers patients with these devastating diseases a mechanism-driven therapeutic option with demonstrated clinical efficacy in reducing proteinuria, stabilizing kidney function, and clearing pathological complement deposits. For Sobi, this approval cements its commitment to serving the rare disease community and reinforces the translational value of targeted complement inhibition in nephrology.