Is Pegcetacoplan legal in the US?
Yes, FDA approved for a rare blood disease and two rare kidney diseases.
AStrong human evidenceFDA approvedas of 2026-10-27
Approved under two brand names. Empaveli, a subcutaneous infusion, was approved in 2021 for paroxysmal nocturnal hemoglobinuria in adults and in July 2025 for C3 glomerulopathy and primary immune-complex membranoproliferative glomerulonephritis in patients 12 and older; it carries a boxed warning for serious infections with encapsulated bacteria. Syfovre, an injection into the eye, was approved in 2023 for geographic atrophy from age-related macular degeneration. 234
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Citations
- Hillmen 2021, N Engl J Med: pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria (PEGASUS) doi.org
- Apellis: Empaveli (pegcetacoplan) prescribing information, revised 2026 pi.apellis.com
- HCPLive 2025: FDA approves pegcetacoplan (Empaveli) for C3 glomerulopathy and IC-MPGN, based on VALIANT hcplive.com
- Zhang 2026, Am J Ophthalmol: endophthalmitis rates after anti-VEGF, corticosteroid implant and anti-complement injections, IRIS Registry pubmed.ncbi.nlm.nih.gov
- Chhablani 2026, Retina: retinal vasculitis events reported to FAERS with intravitreal anti-VEGF and complement inhibitor agents pubmed.ncbi.nlm.nih.gov
Reference information only, not medical advice. Drawn from the Pegcetacoplan article, fact-checked against the cited sources. No dosing anywhere on this site.