In a Phase III trial spanning 124 patients, 122 centers, and 19 countries, pegcetacoplan achieved a 68% reduction in proteinuria, stabilized kidney function, with up to 67% of children in complete remission and 72% showing no disease activity on kidney biopsies.

Foundational research on the complement system by UI professor Richard Smith helped underpin this therapeutic approach and reinforces UI Health Care’s role as a leading treatment center for C3G.

Pegcetacoplan represents a first-of-its-kind therapy that targets the underlying complement system dysfunction driving C3G, rather than merely addressing inflammation.

C3 glomerulopathy (C3G) is a rare pediatric kidney disease that can progress to end-stage kidney failure, affecting about 5,000 Americans with few effective treatments previously available.

Researchers stress translating bench research into therapies and highlight the value of global collaboration in rare-disease trials to demonstrate treatment efficacy.

UI Health Care’s Rare Renal Disease Clinic has become a trusted, globally enrolling center due to comprehensive care, research participation, and patient trust, including international patients.

Pegcetacoplan is given via twice-weekly injections; another complement-targeting drug, iptacopan, was approved earlier for adults with C3G, with UI Health Care leading global enrollment for its trial as well.

The study, led by Carla Nester, MD, at UI Health Care Stead Family Children's Hospital, was published in the New England Journal of Medicine; the FDA had earlier approved pegcetacoplan for patients 12 and older with C3G and related IC-MPGN.

Patient impact is profound, with anecdotes such as a college student in remission and children returning to normal childhood activities, signaling a major prognosis shift for C3G.