#Sweden #Stockholm #Aspaveli #C3G #IC-MPGN

Sobi's Aspaveli® Makes Headway in Treating Rare Kidney Diseases

Sobi® has recently made headlines with a significant announcement regarding its innovative medication, Aspaveli® (pegcetacoplan). The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a favorable opinion recommending the marketing authorization of Aspaveli for treating C3 glomerulopathy (C3G) and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) in adolescents and adults. This critical milestone opens doors to potential treatment options for patients grappling with these rare and severe conditions.

The Need for Effective Treatments

Approximately 8,000 individuals across Europe are currently living with C3G or primary IC-MPGN, rare kidney diseases that pose significant health risks. Tragically, more than half of the individuals diagnosed with these conditions can face kidney failure within five to ten years post-diagnosis, often leading to the necessity of dialysis or life-altering kidney transplants. The emergence of Aspaveli represents hope for these patients, who frequently find themselves with limited treatment options.

CHMP's Endorsement

The positive opinion from the CHMP will now be forwarded to the European Commission, which is anticipated to make a final approval decision in the first quarter of 2026. The endorsement was grounded in the promising results from the Phase 3 VALIANT study. The trial showcased Aspaveli's capacity to significantly reduce proteinuria, stabilize kidney functions, and clear harmful C3 deposits, ultimately leading to enhanced patient outcomes—vital metrics for diseases that tend to progress rapidly. These findings have also been highlighted in a recent publication in The New England Journal of Medicine.

Dr. Lydia Abad-Franch, Sobi’s Chief Medical Officer, shared her enthusiasm regarding this recommendation. "The CHMP's positive opinion for Aspaveli signifies a pivotal moment for individuals affected by C3G or primary IC-MPGN in Europe,” she stated. “With its potential to become the first approved therapy for patients aged 12 and older suffering from these serious kidney ailments, we are excited about the positive impact it could have on patients and their families.”

Insights into C3G and IC-MPGN

C3G and primary IC-MPGN are characterized by excessive C3 deposits in kidney tissues, leading to inflammation, damage, and ultimately, failure of kidney functions. This complicates the health trajectory for those diagnosed as approximately 50% of patients will require significant interventions such as transplants or dialysis within a short window following their diagnosis. Furthermore, there is a notable recurrence risk; studies indicate that nearly 90% of patients who have undergone kidney transplants will see their disease return.

VALIANT Study Details

The VALIANT Phase 3 study was a rigorous, randomized, placebo-controlled trial involving 124 patients aged 12 years and older who suffer from C3G or primary IC-MPGN. This multi-center research marks the largest of its kind, integrating both pediatric and adult subjects from various backgrounds. Over a span of 26 weeks, participants received doses of pegcetacoplan or placebo twice weekly. They were later given the opportunity to continue in an open-label phase, during which all received the active treatment.

About Aspaveli® (pegcetacoplan)

Aspaveli/Empaveli (pegcetacoplan) offers a targeted approach to modulating the complement system, potentially regulating the rampant activation that contributes to severe diseases. This treatment is currently the first approved in the United States for addressing C3G and primary IC-MPGN in patients aged 12 and older, focusing on proteinuria reduction. Moreover, it holds additional approvals for conditions like paroxysmal nocturnal hemoglobinuria (PNH) across various jurisdictions including the U.S. and Europe.

The Sobi® and Apellis® Partnership

Sobi and Apellis have embarked on an international collaboration regarding the development of systemic pegcetacoplan, with Sobi handling the commercial rights outside the U.S. and Apellis overseeing the U.S. market. This partnership underscores the commitment of both companies to provide meaningful advancements in treatment options for patients suffering from rare and critical health conditions.

As Aspaveli moves one step closer to approval in Europe, the hope is that it will transform the lives of thousands who are currently navigating the complexities of C3G and IC-MPGN, bringing much-needed relief and improved quality of life.

For further updates on the approval process and potential availability, stay tuned to official communications from Sobi and health regulatory organizations.