Apellis(APLS) GlobeNewswire·2025-02-20 07:00Core Insights - Apellis Pharmaceuticals and Sobi announced the validation of an indication extension application for Aspaveli (pegcetacoplan) by the European Medicines Agency (EMA) for treating C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), both rare chronic kidney diseases with no approved treatments [1][4] Company and Product Development - The Phase 3 VALIANT study demonstrated a statistically significant 68% reduction in proteinuria in pegcetacoplan-treated patients compared to placebo at Week 26, indicating the drug's efficacy [2][5] - Pegcetacoplan-treated patients also showed stabilization of kidney function and a significant reduction in C3c staining intensity, with favorable safety and tolerability profiles [3][5] - The EMA validation is a significant step towards bringing pegcetacoplan to European patients, with a potential U.S. launch in the second half of 2025 if approved [4][7] Market Context - C3G and IC-MPGN are severe kidney conditions affecting approximately 5,000 people in the U.S. and up to 8,000 in Europe, with a high risk of kidney failure within five to ten years of diagnosis [6] - There is a critical need for approved treatments for these conditions, as 50% of patients may require kidney transplants or lifelong dialysis [6] Collaboration and Commercialization - Apellis and Sobi have global co-development rights for systemic pegcetacoplan, with Sobi holding exclusive commercialization rights outside the U.S. and Apellis holding exclusive rights within the U.S. [8][9] Company Background - Apellis Pharmaceuticals is focused on developing therapies for serious diseases, having introduced the first new class of complement medicine in 15 years, including treatments targeting C3 [10] - Sobi is an international biopharmaceutical company specializing in rare diseases, with a revenue of SEK 26 billion in 2024 [11]