Apellis Reports Robust Clinical Outcomes For EMPAVELI In Rare Kidney Diseases Following NEJM Publication

Apellis Pharmaceuticals announced that The New England Journal of Medicine (NEJM) has published the positive Phase 3 VALIANT study results evaluating EMPAVELI® (pegcetacoplan) for two rare and severe kidney disorders: C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). These findings include both adolescent and adult patient groups, as well as individuals experiencing C3G recurrence after kidney transplantation, marking a significant advancement for conditions with limited therapeutic options.

“The positive data published in the New England Journal of Medicine underscore the unprecedented benefits of EMPAVELI across all three key markers of disease,” said Carla Nester, M.D., MSA, FASN, senior author, professor of internal medicine and pediatrics and director of pediatric nephrology, University of Iowa Stead Family Children's Hospital. “C3G and primary IC-MPGN often lead to kidney failure, and the need for a kidney transplant or dialysis can be devastating. EMPAVELI represents a significant advance in treatment, and I’m thrilled that patients now have access to this important medicine.”  

In the 26-week analysis, EMPAVELI demonstrated effectiveness across all major clinical endpoints. Patients treated with the therapy showed a 68% reduction in proteinuria compared with placebo, alongside stabilization of kidney function with a measurable eGFR benefit. Additionally, the majority of EMPAVELI-treated patients saw dramatic reductions in C3 staining, with 71% achieving complete clearance of C3 deposits—an important indicator of disease control.

“This marks the first time pivotal trial results for a C3G or primary IC-MPGN treatment have been featured in this leading medical journal,” said Peter Hillmen, M.B., Ch.B., Ph.D., chief medical advisor, rare disease, Apellis. “The results show that EMPAVELI has the potential to be life-changing for patients, regardless of their disease type, age, or transplant status. Following the recent FDA approval of EMPAVELI, we are encouraged by the strong response from the nephrology community and growing number of patients starting treatment, and we remain focused on reaching more people living with C3G and primary IC-MPGN.”

The treatment’s safety profile remained consistent with previous studies, showing favorable tolerability. The most commonly reported adverse events in the VALIANT trial included infusion-site reactions, fever, nasopharyngitis, influenza, cough, and nausea. Importantly, across more than 2,750 patient-years of cumulative EMPAVELI exposure, no cases of meningococcal infection related to encapsulated bacteria have been reported.

Longer-term data presented at the European Renal Association Congress and ASN Kidney Week confirmed that EMPAVELI’s benefits were sustained through one year. With FDA approval granted in July 2025 and an EU regulatory decision expected by year-end, EMPAVELI continues to gain momentum as a potential new standard of care for patients with C3G and IC-MPGN.