Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) saw its stock soar 12% after the U.S. Food and Drug Administration (FDA) granted approval for EMPAVELI® (pegcetacoplan), the first approved therapy for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients aged 12 and older.
This approval represents a major advance for individuals affected by these rare kidney disorders, which impact about 5,000 patients in the U.S. and often result in kidney failure. EMPAVELI demonstrated significant benefit in the Phase 3 VALIANT trial, achieving a 68% reduction in proteinuria, kidney function stabilization, and marked clearance of C3 deposits compared to placebo.
“I’m excited to now have a highly effective therapy for a broad range of patients living with C3G and primary IC-MPGN,” said Carla Nester, lead principal investigator for the VALIANT study and professor at the University of Iowa Stead Family Children’s Hospital.
The FDA approval covers a wide patient population, including adults, adolescents, and those experiencing post-transplant recurrence of C3G. This marks Apellis’ third FDA approval within four years, underscoring the company’s dedication to targeting C3-mediated rare diseases.
EMPAVELI’s safety has been well characterized across over 2,200 patient-years in approved uses. The most frequently reported side effects in the VALIANT trial were infusion site reactions, fever, nasopharyngitis, influenza, cough, and nausea.
To support patient access, Apellis has launched the ApellisAssist® program, which helps with treatment navigation, insurance, and financial aid for qualifying patients.
Apellis Pharmaceuticals stock price
This content is for informational purposes only and does not constitute financial, investment, or other professional advice. It should not be considered a recommendation to buy or sell any securities or financial instruments. All investments involve risk, including the potential loss of principal. Past performance is not indicative of future results. You should conduct your own research and consult with a qualified financial advisor before making any investment decisions.