At a glance
| Target | Complement C3 / C3b |
|---|---|
| Systemic product | Empaveli |
| Ophthalmic product | Syfovre |
| Kidney indication age | 12 years and older |
| Systemic major risk | Serious encapsulated bacterial infections |
| Ocular major risk | Retinal vasculitis or vascular occlusion |
Overview
Pegcetacoplan acts relatively early in the complement cascade, affecting both C3-related processes and downstream terminal complement activity. This supports several disease-specific uses. Empaveli is administered subcutaneously for adult paroxysmal nocturnal hemoglobinuria and, under current US labeling, C3 glomerulopathy or primary immune-complex membranoproliferative glomerulonephritis from age 12 to reduce proteinuria and loss of kidney function. Syfovre is a separate eye-injection product for geographic atrophy associated with age-related macular degeneration. A shared active ingredient does not make the products interchangeable. Systemic complement suppression and local ocular administration create different benefits, monitoring needs, and serious risks.
How it works
Pegcetacoplan binds complement C3 and C3b, reducing activation and generation of downstream effectors. In PNH, this can limit both C3-mediated extravascular red-cell destruction and terminal-complement-mediated intravascular hemolysis. In complement-associated kidney disease, it reduces pathway activity implicated in glomerular injury. Local ocular complement inhibition aims to slow geographic-atrophy lesion growth. The mechanism does not restore tissue already lost to retinal degeneration.
Systemic indications and evidence
Empaveli treats adult PNH and specified C3G or primary IC-MPGN patients aged 12 and older. In PNH, the PEGASUS trial showed improved hemoglobin compared with continued eculizumab in selected patients with persistent anemia. Kidney evidence has its own population and outcomes; it should not be inferred solely from PNH results.
What the eye product can do
Syfovre is injected into the eye for geographic atrophy secondary to AMD. Its clinical program showed slower growth of atrophic lesions. This is a disease-progression endpoint, not proof of regenerated retina or restored visual acuity. The eye product is neither a substitute for systemic Empaveli nor an injection for all forms of macular disease.
Supporting sources [2]
Two different safety contexts
Systemic Empaveli carries a boxed warning and REMS for serious infections caused by encapsulated bacteria; vaccination does not eliminate risk. Syfovre warnings include endophthalmitis, retinal detachment, retinal vasculitis or vascular occlusion, neovascular AMD, inflammation, and increased eye pressure. These require formulation-specific assessment rather than one generic safety list.
Why does the kidney indication need current labeling?
Pegcetacoplan’s approved uses have expanded since its original PNH approval. Current Empaveli labeling includes reduction of proteinuria and loss of kidney function for specified C3G and primary IC-MPGN populations. A dated source limited to PNH or only proteinuria can therefore omit a relevant current indication; transplant subgroups still have specific evidence limits.
Supporting sources [1]
Reading this profile
Jurisdiction: United States; indications and availability vary by formulation and country.. Molecular class: PEG-conjugated cyclic peptide complement C3 inhibitor.
This profile was written with AI-assisted research and checked against the linked sources. It has not been represented as a clinician’s review. Evidence summaries describe the cited studies and products; they do not establish that a separately purchased preparation is equivalent.
Sources & references
Follow the original material. Product labels define specific formulations and uses; trial registration describes a study and does not by itself establish benefit.
- Empaveli — current US prescribing information ↗Regulatory source · Checked 2026-10-27
- Syfovre — US prescribing information ↗Regulatory source · Checked 2026-10-27
- Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria. ↗Original research · Checked 2026-10-27