FDA Approves Empaveli for Rare Kidney Diseases

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).

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.

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.

The most common adverse events seen with Empaveli were infusion site reactions, fever, inflammation of the nasal passages and throat, influenza, cough, and nausea. 

“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.”

Approval of Empaveli was granted to Apellis Pharmaceuticals.

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