Real-world efficacy and safety of pegcetacoplan (C3/C3b inhibitor) in 25 pediatric and adult patients with C3 glomerulopathy (C3G), Dense Deposit Disease (DDD), or IC-MPGN who were largely resistant to conventional immunosuppression, evaluated over up to 12 months via uPCR, eGFR, and serum C3.
At 6 months, uPCR fell by 81% (baseline median 3.5 g/g); 68% of patients achieved uPCR <1 g/g, 12% complete remission, and 64% partial remission. eGFR improved by +9.4 ml/min/1.73 m² at month 3 and +12 ml/min/1.73 m² at month 6. Serum C3 normalized or exceeded normal in all but one patient. No serious adverse events occurred over 222 months of cumulative exposure.
- Very small cohort (n=25) from a registry and survey, limiting generalizability. - No control group; observational design precludes causal conclusions. - Sequential biopsy data available in only 4 patients; 12-month follow-up incomplete for some.
For patients with C3G or IC-MPGN resistant to conventional immunosuppression, pegcetacoplan produced rapid, sustained proteinuria reduction and eGFR improvement with no serious adverse events in this real-world cohort. Clinicians should note two potential drug-related concerns flagged in the study and the need for close monitoring, particularly for adherence (tied to C3 normalization).
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