Received U.S. FDA approval for EMPAVELI® (pegcetacoplan) for treatment of patients 12 years and older with C3G and primary IC-MPGNAnnounced capped royalty purchase agreement with Sobi under which Apellis receives up to $300 million for 90% of ex-U.S. royalties of Aspaveli® (pegcetacoplan)Generated $178 million in 2Q 2025 revenues, including $171 million in U.S. net product salesSYFOVRE® (pegcetacoplan injection) injection demand grew 6% quarter-over-quarter, with U.S. net product revenue of $151 millionCash ...

Core Insights - The U.S. FDA has approved EMPAVELI (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients aged 12 and older, aimed at reducing proteinuria [2][3][4] Company Overview - Apellis Pharmaceuticals has achieved its third approval in four years with EMPAVELI, highlighting its focus on targeting C3 to improve patient outcomes [4][27] - The company is committed to supporting patients through programs like ApellisAssist, which offers assistance with insurance coverage, financial aid, and disease education [8][27] Clinical Study Results - The Phase 3 VALIANT study demonstrated a 68% reduction in proteinuria, stabilization of kidney function, and significant clearance of C3 deposits compared to placebo [3][12] - The study included 124 patients aged 12 and older, making it the largest trial for these rare kidney diseases [11] Disease Context - C3G and primary IC-MPGN are rare kidney diseases affecting approximately 5,000 individuals in the U.S., with a high risk of progression to kidney failure [10][12] - About 50% of patients with these conditions may experience kidney failure within five to ten years of diagnosis [10] Safety Profile - EMPAVELI has a well-established safety profile, with over 2,200 patient years of data across approved indications [5][7] - Common adverse reactions (≥10%) include infusion site reactions, pyrexia, and nasopharyngitis [7][25] Future Directions - Apellis plans to advance pivotal studies of EMPAVELI in other rare kidney diseases, building on the momentum from this approval [6][27]

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]