▲ApprovalUS28 Jul 20251 min read
Pegcetacoplan becomes the first approved treatment for two rare kidney diseases
A drug first approved for a blood disorder gains a label for C3 glomerulopathy and primary IC-MPGN, in patients aged 12 and up.
Filed → Approved The molecule is closer to patients than before.
The readA clean example of a peptide drug moving across diseases along a shared pathway. The approval is based on proteinuria, an accepted early marker; the long-term kidney benefit is what doctors will watch.
The decision
The FDA approved pegcetacoplan (Empaveli) for people aged 12 and older with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN). Both are rare kidney diseases in which the complement system, part of innate immunity, damages the kidney’s filters. Until this approval, there was no medicine approved specifically for them.
The evidence
The VALIANT phase 3 trial compared pegcetacoplan with placebo on top of standard care. At week 26, protein in the urine fell by about two thirds more on the drug than on placebo, and kidney function was stable over that period. Biopsies showed less complement deposit in many treated patients.
How the molecule works
Pegcetacoplan is a small cyclic peptide attached to polyethylene glycol, which keeps it in the blood longer. It binds complement protein C3, upstream of where older complement drugs act. Patients infuse it under the skin twice a week with a small pump.
| Point | Detail |
|---|---|
| First approval | Paroxysmal nocturnal hemoglobinuria (PNH), 2021 |
| New use | C3G and primary IC-MPGN, age 12+ |
| Main risk | Serious infections; vaccination required before starting |
Uptake in adolescents, long-term kidney function data and the European decision.
Primary sourceFDA approval and Apellis press release, July 2025; VALIANT phase 3
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