- EMPAVELI (pegcetacoplan) achieves a 68% reduction in proteinuria at Week 26, sustained over one year.
- The study confirms stable kidney function and a favorable safety profile in patients with C3G and IC-MPGN.
- The FDA decision on EMPAVELI (APLS, Financial) is anticipated this summer, with applications under review.
Apellis Pharmaceuticals (APLS) and Sobi have revealed promising findings from their Phase 3 VALIANT study, examining the efficacy of EMPAVELI (pegcetacoplan) in treating C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN)—two rare kidney diseases. The results, presented at the European Renal Association Congress, showed a statistically significant 68% reduction in proteinuria compared to placebo at Week 26, with this effect sustained over a full year.
Beyond the reduction in proteinuria, patients maintained stable kidney function as measured by the estimated glomerular filtration rate (eGFR). Importantly, individuals who transitioned from a placebo to EMPAVELI during the open-label extension phase experienced similar benefits, underscoring the drug's efficacy. EMPAVELI has demonstrated a favorable safety profile, remaining consistent with prior assessments and showing no new safety signals.
The study's insights are particularly significant for patients with C3G and IC-MPGN, who face a high risk of progression to kidney failure. Approximately 50% of these patients develop kidney failure within five to ten years of diagnosis, often requiring a transplant or lifelong dialysis. The promising data from this trial could potentially expand the therapeutic options available to these patients.
The outcome of this study supports the ongoing marketing application reviews with both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Apellis anticipates a decision from the FDA this summer, which will be critical for potentially broadening the market opportunity for EMPAVELI as a treatment for these severe kidney conditions.
