#Sweden #Stockholm #Aspaveli #C3G #Apellis

EMA Validates Aspaveli® Application for Rare Kidney Disease Treatment

The European Medicines Agency (EMA) has approved an indication extension application for Aspaveli® (pegcetacoplan) to treat two critical kidney disorders: C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). This marks a significant milestone for the pharmaceutical companies involved, Sobi® and Apellis Pharmaceuticals, as there are currently no approved treatments for these serious health conditions.

The Urgent Need for Treatment

C3G and IC-MPGN are severe, chronic kidney diseases that can lead to profound health complications, including kidney failure. Patients with these conditions often require life-sustaining treatments such as dialysis or kidney transplants. According to Dr. Lydia Abad-Franch, Chief Medical Officer at Sobi, the validation of this indication extension reflects the commitment to improving outcomes for patients suffering from these rare diseases that affect an estimated 5,000 individuals in the U.S. and up to 8,000 in Europe.

Positive Trial Results

The recent application to the EMA includes positive results from the Phase 3 VALIANT study, which is a randomized, placebo-controlled trial designed to evaluate the efficacy and safety of pegcetacoplan in patients with C3G and IC-MPGN. This study, involving 124 participants aged 12 and older, demonstrated a statistically significant reduction in proteinuria—68% (p<0.0001) in patients treated with pegcetacoplan, compared to those receiving a placebo.

At the 26-week mark, participants displayed stabilization of kidney function as measured by estimated glomerular filtration rate (nominal p=0.03), alongside a notable decline in C3c staining intensity (nominal p<0.0001). Safety and tolerability profiles were consistent with previously established results for pegcetacoplan.

Collaborative Efforts to Advance Treatment

Dr. Jeffrey Eisele, Chief Development Officer at Apellis, emphasized the urgent need for a treatment that can help sustain kidney function for patients facing these conditions. The validation from the EMA is an essential step toward making this therapy available to European patients. Additionally, the companies are in the process of furthering the regulatory approval in the United States, with hopes for a potential launch in the latter half of 2025.

About C3G and IC-MPGN

C3G and IC-MPGN are rare kidney diseases characterized by the accumulation of C3c deposits, leading to inflammation and damage to kidney tissues, ultimately resulting in kidney failure in many cases. Patients diagnosed with these diseases face a daunting prognosis, with about 50% experiencing kidney failure within five to ten years post-diagnosis. Most patients who have undergone a kidney transplant also see disease recurrence, presenting an ongoing public health challenge.

Innovation in Treatment: Pegcetacoplan

Pegcetacoplan represents a targeted approach to managing the complement system—an integral part of the immune response that can contribute to disease pathogenesis. It is currently studied for various rare diseases within nephrology and hematology fields. Pegcetacoplan is already approved for treating paroxysmal nocturnal hemoglobinuria (PNH) under the brand names Empaveli® and Aspaveli® in multiple markets, showcasing its potential versatility.

Sobi and Apellis Collaboration

The joint efforts of Sobi and Apellis underscore the commitment to addressing unmet medical needs in rare diseases. While Sobi maintains exclusive commercial rights for systemic pegcetacoplan outside the United States, Apellis has exclusive rights for the U.S. market. Together, these companies aim to bring groundbreaking therapies to patients who have long waited for effective treatment options.

In conclusion, the EMA's validation of the Aspaveli® application signifies hope for patients battling C3G and IC-MPGN, not only enhancing clinical outcomes but also paving the way for future innovations within the realm of rare diseases.

For additional details on these companies, please visit Sobi and Apellis websites.