Approval is first treatment for C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis<\/p>\n<\/h3>\n
By Lori Solomon HealthDay Reporter<\/b>
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FRIDAY, Aug. 1, 2025 (HealthDay News) — The U.S. Food and Drug Administration has approved Empaveli (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).<\/p>\n
The drug has been approved for patients 12 years and older and works to reduce proteinuria with these rare diseases that affect the filtering units in the kidneys.<\/p>\n
The approval is supported by the phase 3 VALIANT study, in which patients taking Empaveli had a 68 percent reduction in proteinuria, a stabilization of kidney function, and a substantial clearance of C3 deposits (measured by C3 staining) compared with placebo. Among Empaveli-treated patients, 71 percent achieved zero C3 staining intensity, showing complete clearance of C3 deposits. The results were consistent across adolescent and adult patients for both C3G and primary IC-MPGN, as well as in C3G patients with posttransplant disease recurrence.<\/p>\n
The most common adverse events seen with Empaveli were infusion site reactions, fever, inflammation of the nasal passages and throat, influenza, cough, and nausea. <\/p>\n
“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,” Carla Nester, M.D., lead principal investigator for the VALIANT study from the University of Iowa in Iowa City, said in a statement. “Given the urgent need, particularly in children, the approval of Empaveli marks a pivotal moment in the treatment of rare kidney diseases.”<\/p>\n
Approval of Empaveli was granted to Apellis Pharmaceuticals.<\/p>\n