Apellis Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review designation of the supplemental New Drug Application (sNDA) for EMPAVELI (pegcetacoplan) for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), which are severe and rare kidney diseases. The Prescription Drug User Fee Act (PDUFA) target action date is July 28, 2025.
“EMPAVELI demonstrated clinically meaningful benefits across all three key markers of disease – unprecedented proteinuria reductions, stabilization of kidney function, and substantial clearance of C3c staining,” said Bradley P. Dixon, M.D., FASN, professor of pediatrics and medicine, University of Colorado School of Medicine, and co-investigator of the VALIANT study. “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 has the potential to make a life-changing difference for patients, if approved.”
“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,” said Cedric Francois, M.D., Ph.D., chief executive officer and co-founder at Apellis. “We look forward to working with the FDA to make this treatment available to patients in need as quickly as possible.”
sNDA submission supported by positive Phase 3 VALIANT results at Week 26
The positive results were consistent across patients with C3G and IC-MPGN, adolescents and adults, and native and post-transplant kidney disease.
- Proteinuria reduction: The study met its primary endpoint, demonstrating a statistically significant 68% (p<0.0001) proteinuria reduction in EMPAVELI-treated patients compared to placebo.
- Stabilization of kidney function: EMPAVELI-treated patients achieved stabilization of kidney function (nominal p=0.03) as measured by eGFR.
- Reduction of C3c staining: A substantial proportion of EMPAVELI-treated patients achieved a reduction in C3c staining intensity (nominal p<0.0001). 71% of EMPAVELI-treated patients showed complete clearance of C3c staining compared to placebo.
EMPAVELI showed favorable safety and tolerability, consistent with its established profile.
Priority Review designation is granted to marketing applications for medicines that treat a serious condition and if approved, would provide a significant improvement in the safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition.