Carla Nester, MD, Professor of Pediatrics-Nephrology at the University of Iowa, discusses one-year phase 3 data of Empaveli (pegcetacoplan) for treatment of C3 glomerulopathy (C3G) and immune complex membranoproliferative glomerulonephritis (IC-MPGN).
C3G and IC-MPGN are rare kidney diseases characterized by damage to the glomeruli in the kidney. It is believed to be caused by the accumulation of proteins in the kidney due to abnormal activation of the complement system due to genetic mutations or the absence of regulatory proteins. Common signs and symptoms include hematuria, proteinuria, reduced glomerular filtration rate and increased creatinine levels, fatigue, and edema of the hands, feet, and ankles.
One-year phase 3 data was recently presented at the American Society of Nephrology (ASN) Kidney Week 2025 that reinforces the robust and sustained efficacy of pegcetacoplan for C3G and IC-MPGN. Pegcetacoplan is a targeted C3 therapy designed to regulate the excessive activation of the complement cascade. It was approved by the U.S. Food and Drug Administration (FDA) in July 2025 for the treatment of patients ages 12 years and older with C3G or primary IC-MPGN.
Data from the phase 3 study showed maintained proteinuria reduction of 68% achieved at week 26, regardless of immunosuppressant use or baseline proteinuria levels. Complete proteinuria remission was achieved in one-third of patients and sustained through one year, compared to 3% for placebo at week 26. Additionally, pegcetacoplan continued to stabilize kidney function as manifested by estimated glomerular filtration rate (eGFR) and its safety profile remained well-tolerated and consistent.
Dr. Nester also noted that, two anchored indirect treatment comparisons showed pegcetacoplan was superior to iptacopan in patients with C3G in reducing proteinuria and achieving the composite renal endpoint, which combines proteinuria reduction and stabilization of eGFR. A significantly greater proportion of pegcetacoplan-treated patients achieved urine protein creatinine ratio (UPCR) reduction to less than 1 g/g, at least a 50% reduction in UPCR, and the composite renal endpoint.
Read the results of VALIANT clinical trial in the New England Journal of Medicine.
For more information, click here.
To learn more about C3G, IC-MPGN, and other rare kidney diseases, visit https://checkrare.com/diseases/kidney-and-urinary-diseases/