Apellis and Sobi Announce EMPAVELI® (pegcetacoplan) Showed Sustained Efficacy at One Year in Phase 3 Study for C3G and Primary IC-MPGN

Core Insights - Apellis Pharmaceuticals and Sobi presented new data from the Phase 3 VALIANT study for EMPAVELI (pegcetacoplan) targeting C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) at the European Renal Association Congress [1][5] Study Results - EMPAVELI showed a statistically significant 68% reduction in proteinuria compared to placebo at Week 26, with effects sustained for one year [2][4] - Patients switching from placebo to EMPAVELI also experienced similar benefits in proteinuria reduction and kidney function stabilization [3] Efficacy and Safety - The study confirmed EMPAVELI's efficacy and safety profile across a diverse patient population, including adults and adolescents with both native and post-transplant kidney disease [4][7] - No new safety signals were reported, indicating favorable safety and tolerability consistent with its established profile [4][7] Clinical Significance - The results highlight the urgent need for effective treatments for patients with C3G and primary IC-MPGN, as approximately 50% of these patients may face kidney failure within five to ten years of diagnosis [6] - The VALIANT study is the largest trial conducted in these populations, involving 124 patients aged 12 and older [8] Future Prospects - Marketing applications for EMPAVELI are currently under review with the FDA and EMA, with an FDA decision expected this summer [4][7] - The collaboration between Apellis and Sobi aims to expedite the availability of EMPAVELI for patients suffering from these rare kidney diseases [10]