FDA Approves Apellis’ EMPAVELI® (pegcetacoplan) as the First C3G and Primary IC-MPGN Treatment for Patients 12 and Older

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]