FDA Approves Apellis’ Empaveli for C3G and Primary IC-MPGN for Patients 12 and Older

Rare Daily Staff

The U.S. Food and Drug Administration has approved Apellis Pharmaceuticals’ Empaveli as the first approved treatment for the rare kidney diseases C3 glomerulopathy and first approved treatment primary immune complex membranoproliferative glomerulonephritis in patients 12 years of age and older, to reduce proteinuria.

C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) can lead to kidney failure. Excessive C3 deposits are a key marker of disease activity, which can result in kidney inflammation, damage, and eventual failure. Up to 50 percent of individuals with C3G and primary IC-MPGN progress to kidney failure within five to ten years of diagnosis, often requiring kidney transplantation or lifelong dialysis therapy. Additionally, up to approximately 90 percent of patients who previously received a kidney transplant for these conditions experience disease recurrence.

Empaveli is a targeted C3 inhibitor designed to regulate excessive activation of the complement cascade, which is part of the body’s immune system and associated with the onset and progression of many serious diseases. It is approved for the treatment of C3G and primary IC-MPGN in the United States, and for paroxysmal nocturnal hemoglobinuria  in the United States, European Union, and other countries globally. The therapy is also under investigation for other rare diseases.

Approval was supported by positive six-month results from the phase 3 VALIANT study, which demonstrated benefits across multiple disease markers. The study met its primary endpoint, showing a statistically significant 68 percent reduction in proteinuria in Empaveli-treated patients compared to placebo, along with stabilization of kidney function as measured by estimated glomerular filtration rate.

The most common adverse reactions observed in the VALIANT study were infusion site reactions, pyrexia, nasopharyngitis, 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,” said Carla Nester, lead principal investigator for the VALIANT study, professor of internal medicine and pediatrics, and director of pediatric nephrology at the University of Iowa Stead Family Children’s Hospital. “Given the urgent need, particularly in children, the approval of Empaveli marks a pivotal moment in the treatment of rare kidney diseases.”