Apellis Gains FDA Nod for C3G and Primary IC-MPGN Treatment
Pegcetacoplan (EMPAVELI), which was shown to be safe and effective in a Phase III clinical trial, is now approved for the treatment of U.S. patients 12 years and older with C3G and primary IC-MPGN.
The U.S. FDA has approved Apellis Pharmaceuticals’ pegcetacoplan (EMPAVELI) as a treatment for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients aged 12 years or older. This regulatory decision, which was announced by Apellis in a July 28, 2025 press release (1), marks the first for the rare kidney diseases, meeting an unmet clinical need.
“The approval of EMPAVELI is a historic milestone for people living with C3G and primary IC-MPGN, many of whom are adolescents or young adults,” said Josh Tarnoff, Chief Executive Officer, NephCure, in the press release (1). “We recognize Apellis’ commitment to these patients and their families, and to the research and innovation that will bring this life-changing treatment into the hands of patients that need it most.”
C3G and primary IC-MPGN are rare kidney diseases that affect approximately 5,000 people in the U.S. and 8,000 people in Europe. A key marker of the disease activity is excessive C3 deposits, which can lead to kidney inflammation, damage, and failure. Approximately half of those who suffer from C3G and primary IC-MPGN experience kidney failure within five to 10 years after being diagnosed.
“EMPAVELI has the potential to be truly transformational for patients with C3G and primary IC-MPGN, who until now have had very few treatment options. In the largest pivotal study of these diseases, EMPAVELI demonstrated its potential to preserve kidney function by controlling all three key markers of disease,” added Cedric Francois, M.D., Ph.D., Co-Founder and Chief Executive Officer, Apellis, in the press release (1). “As Apellis’ third approval in four years, this milestone underscores the unique ability of targeting C3 to improve patients’ lives. We are deeply grateful to everyone who made this approval possible and look forward to building on this momentum as we advance pivotal studies of EMPAVELI in other rare kidney diseases.”
“I’m excited to now have a highly effective therapy for a broad range of patients living with C3G and primary IC-MPGN,” commented Carla Nester, M.D., MSA, FASN, Lead Principal Investigator for the VALIANT study, Professor of Internal Medicine and Pediatrics and Director of Pediatric Nephrology, University of Iowa Stead Family Children's Hospital, in the press release (1). “With standard of care, patients living with these rare and severe diseases frequently progress to kidney failure, necessitating lifelong dialysis and/or a kidney transplant. Given the urgent need, particularly in children, the approval of EMPAVELI marks a pivotal moment in the treatment of rare kidney diseases.”
FDA’s decision was based on positive six-month results of the Phase III VALIANT clinical trial, which demonstrated a 68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits as measured by C3 staining, compared to placebo. Additionally, the safety profile of pegcetacoplan is well-established and no new safety signals were found during the trial (2).
“The one-year Phase III results are very compelling, confirming EMPAVELI’s sustained benefits across key markers of disease,” said Fadi Fakhouri, M.D., Ph.D., Presenting Author, Co-Lead Principal Investigator for the VALIANT study, and Professor of Nephrology at CHUV Lausanne, Switzerland, in a press release about the study results (2). “Given the high risk of kidney failure, treatment efficacy is incredibly important to C3G and primary IC-MPGN patients, many of whom are in the prime of their lives. These data further underscore the potential of EMPAVELI to make a meaningful difference for patients.”
References
Apellis Pharmaceuticals. FDA Approves Apellis’ EMPAVELI (Pegcetacoplan) as the First C3G and Primary IC-MPGN Treatment for Patients 12 and Older. Press Release, July 28, 2025.
Apellis Pharmaceuticals. Apellis and Sobi Announce EMPAVELI (Pegcetacoplan) Showed Sustained Efficacy at One Year in Phase III Study for C3G and Primary IC-MPGN. Press Release, June 6, 2025.