Breakthroughs in Treating Rare Kidney Diseases: Pegcetacoplan and Beyond

Pegcetacoplan pediatric trial readouts and recent regulatory action position the drug as a potential practice-changing therapy for children with C3 glomerulopathy (C3G). The trial reported a 68% reduction in proteinuria and up to 67% complete remission, with stabilization of kidney function—findings that may alter management for children with active disease.

The randomized phase III pediatric study enrolled children with C3G and used proteinuria reduction and preservation of kidney function as primary endpoints. Investigators reported the outcomes above and noted no new major safety signals beyond expected injection-site reactions and complement-related effects.

Protocol changes may include earlier referral and consideration of targeted complement blockade for children with persistent high proteinuria despite optimized supportive therapy. Pegcetacoplan would be integrated alongside—not replace—current measures: blood-pressure control, RAAS blockade, and selective immunosuppression. Successful incorporation will require alignment of dosing schedules and laboratory monitoring for complement activity and infection risk.

Regulatory approval and demonstrated pediatric efficacy will likely concentrate referrals to experienced centers, increasing demand for specialty services and affecting access logistics such as travel, insurance authorization, and clinic capacity for administration and monitoring.