FDA Approves Pegcetacoplan for C3G and Primary IC-MPGN in Patients 12 and Older
BOSTON, MA – July 28, 2025 – Apellis Pharmaceuticals announced today the U.S. Food and Drug Administration (FDA) has approved EMPAVELI (pegcetacoplan) as the first treatment for patients aged 12 and older diagnosed with C3 glomerulopathy (C3G) and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN). This landmark approval marks a meaningful advancement in the treatment landscape for these rare kidney diseases.
The approval is based on the results of the Phase III VALIANT study, which demonstrated EMPAVELI’s efficacy and safety. In the trial, 124 patients were randomized to recieve either pegcetacoplan or a placebo twice weekly for 26 weeks. Of these, 63 patients received pegcetacoplan, and 61 received a placebo. Following the blinded phase, all participants were offered the opportunity to enroll in a subsequent 26-week open-label phase, during which all patients received pegcetacoplan.
the VALIANT study met its primary endpoint, showing a statistically significant 68% reduction in urinary protein-to-creatinine ratio (UPCR) in patients treated with pegcetacoplan compared to placebo at week 26 (P < .0001). Furthermore, pegcetacoplan treatment led to the stabilization of kidney function, as measured by estimated glomerular filtration rate (eGFR), with a nominal P-value of .03. The therapy also resulted in a ample reduction in C3c staining intensity, with 71% of patients achieving complete clearance compared to the placebo group (nominal P < .0001). Researchers observed that the benefits of pegcetacoplan were consistent across various patient subgroups, including those with C3G and primary IC-MPGN, adolescents and adults, and patients with either native or post-transplant kidney disease. The safety and tolerability profile of pegcetacoplan was found to be favorable and consistent with previously reported data. "EMPAVELI demonstrated clinically meaningful benefits across all 3 key markers of disease - unprecedented proteinuria reductions,stabilization of kidney function,and substantial clearance of C3c staining," stated Bradley P. Dixon, MD, FASH, professor of pediatrics and medicine at the University of Colorado School of Medicine and VALIANT co-investigator. "As a disease-modifying therapy, EMPAVELI has the potential to make a life-changing difference for patients." References:
- FDA approves Apellis’ EMPAVELI (pegcetacoplan) as the first C3G and primary IC-MPGN treatment for patients 12 and older. News release. Apellis. July 28, 2025. Accessed July 28, 2025.
- Apellis announces FDA acceptance and priority review of the supplemental new drug request for EMPAVELI (pegcetacoplan) for C3G and primary IC-MPGN. News release. Apellis. April 1, 2025. Accessed July 28, 2025.
- Phase III study assessing the efficacy and safety of pegcetacoplan in patients with C3 glomerulopathy or immune-complex membranoproliferative glomerulonephritis (VALIANT). ClinicalTrials.gov. Updated March 13, 2025. Accessed July 28,2025.
