FDA Approves Apellis' EMPAVELI® (pegcetacoplan) as the First C3G and Primary IC-MPGN Treatment for Patients 12 and Older

Proven efficacy across all three key markers of disease—68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 depositsBroad label includes adults and adolescents with C3G or primary IC-MPGN, and post-transplant C3G disease recurrenceWell-established safety profile, consistent across >2,200 patient years in approved indicationsC3G and primary IC-MPGN are rare kidney diseases with high risk of kidney failureConference call tomorrow at 8:00 a.m. ETWALTHAM, Mass., July 28, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) today announced that the U.S. Food and Drug Administration (FDA) has approved EMPAVELI® (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older, to reduce proteinuria. C3G and primary IC-MPGN are rare kidney diseases, affecting 5,000 people in the United States.1"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, M.D., MSA, FASN, lead principal investigator for the VALIANT study, professor of internal medicine and pediatrics and director of pediatric nephrology, University of Iowa Stead Family Children's Hospital. "With standard of care, patients living with these rare and severe diseases frequently progress to kidney failure, necessitating lifelong dialysis and/or a kidney transplant. Given the urgent need, particularly in children, the approval of EMPAVELI marks a pivotal moment in the treatment of rare kidney diseases."In the Phase 3 VALIANT study, EMPAVELI demonstrated an unprecedented 68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits as measured by C3 staining, compared to placebo. The positive results were consistent across adolescent and adult patients with C3G and primary IC-MPGN, and in C3G patients with post-transplant disease recurrence."EMPAVELI has the potential to be truly transformational for patients with C3G and primary IC-MPGN, who until now have had very few treatment options. In the largest pivotal study of these diseases, EMPAVELI demonstrated its potential to preserve kidney function by controlling all three key markers of disease," said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer, Apellis. "As Apellis' third approval in four years, this milestone underscores the unique ability of targeting C3 to improve patients' lives. We are deeply grateful to everyone who made this approval possible and look forward to building on this momentum as we advance pivotal studies of EMPAVELI in other rare kidney diseases.""The approval of EMPAVELI is a historic milestone for people living with C3G and primary IC-MPGN, many of whom are adolescents or young adults," said Josh Tarnoff, chief executive officer, NephCure. "We recognize Apellis' commitment to these patients and their families, and to the research and innovation that will bring this life-changing treatment into the hands of patients that need it most."The approval of EMPAVELI is based on positive six-month results from the VALIANT study, demonstrating benefits across all three key markers of disease:Proteinuria reduction: The study met its primary endpoint, demonstrating a statistically significant 68% (pStabilization of kidney function: EMPAVELI-treated patients achieved stabilization of kidney function compared to placebo (nominal p=0.03) as measured by estimated glomerular filtration rate (eGFR).Reduction of C3 staining: A majority of EMPAVELI-treated patients achieved a reduction in C3 staining intensity (nominal pThe safety profile of EMPAVELI is well-established, with >2,200 patient years across all approved indications. The most common adverse reactions in the VALIANT study (≥10%) were infusion site reactions, pyrexia, nasopharyngitis, influenza, cough, and nausea. Apellis is committed to helping patients with treatment access and support. ApellisAssist® is a program designed to help EMPAVELI patients along their treatment journey by providing a comprehensive system of support including help navigating insurance coverage, financial assistance for eligible patients, disease education, and ongoing product support. Patients and healthcare providers can call 1-888-273-5547 for more information.Conference Call and WebcastApellis will host a conference call and webcast to discuss the FDA's approval of EMPAVELI tomorrow, July 29, 2025 at 8:00 a.m. ET. To access the live call by phone, please pre-register for the call here. A live audio webcast of the event and accompanying slides may also be accessed through the "Events and Presentations" page of the "Investors and ...Full story available on Benzinga.com