EC Validates Aspaveli Extension

“C3G and IC-MPGN are severe and life-threatening kidney conditions, often leading to kidney failure and requiring a kidney transplant or dialysis for life,” said Lydia Abad-Franch MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. “With no approved treatments currently available, this important milestone reflects our dedication to improving outcomes for those affected by these rare kidney conditions.”

The submission includes positive data from the Phase III VALIANT study. The study met its primary endpoint demonstrating a statistically significant and clinically meaningful 68% (p<0.0001) proteinuria reduction in pegcetacoplan-treated patients compared to placebo. At week 26, results were consistent agross subgroups, including disease type, age, and transplant status.Additionally, pegcetacoplan-treated patients achieved stabilisation of kidney function (nominal p=0.03), as measured by estimated glomerular filtration rate, and a substantial proportion of patients achieved a reduction in C3c staining intensity (nominal p<0.0001). Pegcetacoplan showed favourable safety and tolerability, consistent with its established profile.

“There is an urgent need for an approved treatment for C3G and IC-MPGN that can prolong kidney function,” said Jeffrey Eisele, Ph.D., Chief Development Officer at Apellis. The EMA validation leads us one step closer to potentially bringing this treatment to European patients in need. Additionally, we continue to advance the regulatory process in the U.S., with a potential launch of pegcetacoplan for C3G and IC-MPGN in the second half of 2025, if approved.”