Sobi Nears Finish Line with Kidney Drug, Offering Hope for Thousands

STOCKHOLM, SWEDEN – December 12, 2025 – In a move that signals a potential paradigm shift for thousands of patients facing kidney failure, Swedish biopharma company Sobi announced it has received a positive opinion from a key European regulatory committee for its drug Aspaveli® (pegcetacoplan). The recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) is for the treatment of two rare and devastating kidney diseases, C3 glomerulopathy (C3G) and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN), in patients aged 12 and older.

This decision puts Sobi on the cusp of delivering the first-ever approved therapy in Europe specifically designed to combat the underlying cause of these conditions. For the approximately 8,000 people in Europe living with the daily threat of kidney failure from these diseases, this news represents more than just a regulatory milestone; it represents a tangible beacon of hope.

The Crushing Burden of C3G and IC-MPGN

To understand the significance of the CHMP's opinion, one must first grasp the grim reality for those diagnosed with C3G and primary IC-MPGN. These aren't chronic conditions that can be easily managed; they are aggressive diseases, particularly affecting adolescents and young adults, that relentlessly attack the kidneys. The root cause is a malfunction in the complement cascade, a part of the immune system. This dysregulation leads to excessive deposits of a protein called C3 in the kidneys, triggering inflammation and progressive, irreversible damage.

The current standard of care is a frustrating patchwork of supportive treatments and broad immunosuppressants, like steroids, which come with their own significant side effects and often fail to halt the disease's progression. The prognosis is stark: roughly half of all patients will suffer complete kidney failure within five to ten years of diagnosis. This forces them into a life of burdensome dialysis or the pursuit of a kidney transplant.

Even transplantation is not a cure. The underlying complement system dysfunction persists, and in approximately 90% of cases, the disease returns to attack the new, transplanted kidney. This high recurrence rate creates a devastating cycle of hope and despair for patients and their families, highlighting the profound unmet need for a therapy that targets the disease mechanism itself, not just its symptoms.

Unlocking the Complement System: The Science Behind Aspaveli

The promise of Aspaveli lies in its precision. As a targeted C3 inhibitor, pegcetacoplan is designed to regulate the overactivation of the complement cascade at its source. By binding to and controlling C3 and its fragments, the therapy aims to prevent the protein deposits that drive kidney inflammation and damage. This approach moves treatment from broad suppression to targeted intervention.

The CHMP's recommendation was not made lightly; it is built on the robust foundation of the Phase 3 VALIANT study, the largest clinical trial ever conducted in this patient population. The study, whose results were recently published in the prestigious New England Journal of Medicine, demonstrated that Aspaveli achieved a trifecta of clinically meaningful outcomes.

First, it met its primary endpoint with a statistically significant 68% reduction in proteinuria (excess protein in the urine, a key marker of kidney damage) compared to placebo. Second, it demonstrated a stabilization of kidney function, a critical measure for preventing progression to end-stage renal disease. Finally, and perhaps most impressively, it led to the substantial or complete clearance of the disease-causing C3 deposits in the kidneys of a majority of patients. It is the only therapy to have shown significant benefits across all three of these key markers of disease.

"The CHMP's positive opinion 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 kidney failure," stated Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. "If approved, Aspaveli would become the first therapy for patients 12 years and older with these serious kidney diseases, which often affect adolescents and young adults."

From Regulatory Opinion to Market Reality

While the positive CHMP opinion is a monumental hurdle cleared, the journey to patient access is not yet complete. The recommendation will now be referred to the European Commission (EC), which will make the final decision on marketing authorization, expected in the first quarter of 2026. An EC approval would grant a unified marketing authorization across the European Union.

Following that, Sobi will face the complex, country-by-country process of securing reimbursement. This involves negotiating with national health technology assessment (HTA) bodies, which scrutinize a new drug's clinical efficacy and cost-effectiveness before recommending it for public funding. This stage is a critical battleground where clinical innovation meets economic reality, and it will determine how quickly and widely Aspaveli becomes available to the patients who need it.

For investors and healthcare executives, this pathway is a key area to watch. Sobi's ability to effectively communicate Aspaveli's value proposition—its potential to not only improve quality of life but also to offset the immense long-term costs of dialysis and transplantation—will be crucial for its commercial success in Europe.

A Strategic Win for Sobi and Its Partnership Model

This regulatory progress is also a significant strategic victory for Sobi, reinforcing its position as a leader in the lucrative but challenging rare disease market. The company's collaboration with Apellis Pharmaceuticals, which holds U.S. commercialization rights, is a textbook example of leveraging partnership to de-risk and accelerate development. Under their agreement, Sobi has exclusive commercialization rights outside the United States.

The potential revenue stream is substantial. While pricing in Europe is yet to be determined, rare disease therapies with first-in-class status command premium prices. Analysts have already projected peak U.S. sales for the drug in these indications could reach $700 million, signaling a significant global market opportunity. For Sobi, whose 2024 revenue was SEK 26 billion, a successful European launch for Aspaveli in this new indication would provide a powerful new growth driver.

The partnership's financial structure, which includes milestone payments and royalties, reflects a shared confidence in the drug's potential. A recent royalty purchase agreement further aligned the companies, demonstrating Sobi's commitment to maximizing the value of its ex-U.S. rights. As Sobi prepares its commercial infrastructure for a potential 2026 launch, the successful execution of this strategy will be a key determinant of its future growth and its ability to continue funding a pipeline of innovative therapies for other rare conditions.