FDA Approves First Treatment for Rare Kidney Diseases C3G, IC-MPGN

The U.S. Food and Drug Administration (FDA) has approved EMPAVELI® (pegcetacoplan) from Apellis Pharmaceuticals as the first treatment for the rare and severe kidney diseases C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients aged 12 and older. These diseases affect approximately 5,000 individuals in the U.S. and have a high risk of progression to kidney failure, often requiring dialysis or transplant, with about 90% of transplant recipients experiencing disease recurrence. EMPAVELI demonstrated a 68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits in the Phase 3 VALIANT trial, with consistent efficacy across adolescent and adult patients, including those with post-transplant recurrence. While the drug offers a significant advancement in treatment options, it requires a Risk Evaluation and Mitigation Strategy (REMS) due to an increased risk of serious infections caused by encapsulated bacteria, necessitating appropriate vaccinations before treatment. The approval broadens therapeutic choices for patients with high unmet needs and positions Apellis Pharmaceuticals for potential revenue growth in the nephrology market. Experts highlight the approval as a pivotal moment, especially for pediatric patients living with these challenging diseases.