Apellis’ EMPAVELI® Shows Strong Phase 3 Results, Gains FDA Priority Review for C3G and IC-MPGN

Apellis Pharmaceuticals announced that the FDA has granted Priority Review to the sNDA for EMPAVELI® (pegcetacoplan) to treat C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), rare and severe kidney diseases. The PDUFA target date for a decision is July 28, 2025. The FDA’s Priority Review designation accelerates the review process for drugs that could significantly improve treatment for severe conditions.

Bradley P. Dixon, M.D., FASN, professor of paediatrics and medicine, University of Colorado School of Medicine, and co-investigator of the VALIANT study, stated, “EMPAVELI demonstrated clinically meaningful benefits across all three key markers of disease – unprecedented proteinuria reductions, stabilisation of kidney function, and substantial clearance of C3c staining. Efficacy is critically important when treating C3G and IC-MPGN given the high risk of progression to kidney failure. As a disease-modifying therapy, EMPAVELI can potentially make a life-changing difference for patients, if approved.”

The sNDA is backed by positive Phase 3 VALIANT study results, which showed 68% proteinuria reduction (p<0.0001) compared to the placebo. The results also showed the stabilisation of kidney function (p=0.03) along with 71% clearance of C3c staining and exhibited favourable safety and tolerability in line with previous studies.

“This milestone represents a significant step toward our goal of bringing EMPAVELI to people living with C3G and primary IC-MPGN, regardless of their disease type, age, or transplant status. We look forward to working with the FDA to make this treatment available to needy patients as quickly as possible,” mentioned Cedric Francois, M.D., Ph.D., chief executive officer and co-founder at Apellis.