A Phase III trial of the complement inhibitor pegcetacoplan in C3 glomerulopathy (C3G) reported striking results in the New England Journal of Medicine: the drug produced a 68% reduction in urine protein and high rates of remission and biopsy improvement in pediatric and young adult patients. The multicenter, randomized, placebo‑controlled study was led by Carla Nester at University of Iowa and enrolled patients across 19 countries. Pegcetacoplan, already approved earlier this year for patients 12 and older with C3G and IC‑MPGN, directly targets complement dysregulation — the root cause of disease pathology — and the trial data strengthen its position as a disease‑modifying option in very rare glomerular disease.