Apellis And Sobi Report EMPAVELI Sustains 68% Proteinuria Reduction At 1 Year In Rare Kidney Diseases

Apellis Pharmaceuticals and Swedish Orphan Biovitrum (Sobi) presented new data from the open-label phase of the Phase 3 VALIANT study evaluating EMPAVELI® (pegcetacoplan) in patients with C3 glomerulopathy (C3G) and immune complex membranoproliferative glomerulonephritis (IC-MPGN). 

“The one-year Phase 3 results are very compelling, confirming EMPAVELI’s sustained benefits across key markers of disease. 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,” 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. 

EMPAVELI achieved a 68% reduction in proteinuria compared to placebo at Week 26, a result that was sustained through one year of treatment. Patients switching from placebo to EMPAVELI during the open-label period also saw comparable improvements in proteinuria and maintained kidney function, as measured by estimated glomerular filtration rate (eGFR).

“These data reinforce the strength of the EMPAVELI efficacy and safety profile across a broad population of patients with C3G and primary IC-MPGN, including adults and adolescents with native and post-transplant kidney disease. With an FDA decision this summer, we look forward to bringing EMPAVELI to patients living with these rare and severe kidney diseases as quickly as possible,” said Peter Hillmen, M.B., Ch.B., Ph.D., chief medical advisor, rare disease, Apellis.

The treatment continued to show a favorable safety and tolerability profile, with no new safety signals observed. These results reinforce EMPAVELI’s promise as a potential therapy for rare and serious kidney diseases marked by complement dysregulation.

“The results from the Phase 3 VALIANT study underscore the potential of EMPAVELI in addressing the urgent needs of patients living with the kidney diseases C3G and primary IC-MPGN. These studies are an example of Sobi’s commitment to advance innovative therapies that make a meaningful difference in patients' lives,” said Nils Kinnman, M.D., Ph.D., head of medical affairs and clinical development, Sobi.