Pegcetacoplan
Also known as: APL-2, Empaveli, Syfovre
Pegcetacoplan inhibits complement C3. Its subcutaneous Empaveli and intravitreal Syfovre products serve different diseases and have distinct infection, eye and discontinuation risks.
Quick Facts
One molecule, two product settings
Pegcetacoplan is a pegylated cyclic peptide, developed as APL-2, that binds complement protein C3 and activation fragment C3b. C3 is central to innate-immunity complement signaling. Empaveli is a subcutaneous product for systemic diseases, while Syfovre is injected into the eye for geographic atrophy. These routes and uses must not be conflated.
Complement inhibition and PEGASUS
C3 blockade interrupts downstream complement-mediated cell injury. In paroxysmal nocturnal hemoglobinuria, red cells lack protective surface proteins and are destroyed both within vessels and through C3-mediated surface effects. Acting at C3 addresses both pathways, broader than inhibition of the later C5 step alone. PEGASUS found pegcetacoplan superior to C5 inhibitor eculizumab for hemoglobin improvement over 16 weeks.
Complement overactivity is also implicated in retinal-cell loss in geographic atrophy. Intravitreal treatment is intended to slow that progression; it is a separate clinical context from controlling hemolysis.
Supporting references: superior.
Sources: [1]
Product-specific FDA approvals
| Year | Product | Indication |
|---|---|---|
| 2021 | Empaveli | Paroxysmal nocturnal hemoglobinuria |
| 2023 | Syfovre | Geographic atrophy secondary to age-related macular degeneration |
| 2025 | Empaveli | C3 glomerulopathy and primary immune-complex membranoproliferative glomerulonephritis |
Supporting references: approved.
Sources: [2]
Who receives each formulation
Empaveli treats adults with PNH and reduces proteinuria in people aged 12 or older with C3 glomerulopathy or primary IC-MPGN. Syfovre is administered by an ophthalmologist into the eye for geographic atrophy, an advanced dry age-related macular degeneration setting. Empaveli carries a boxed warning for serious encapsulated-bacterial infection, requiring vaccination and a restricted risk-management program.
Systemic and eye risks
Empaveli’s common effects include injection reactions, infections, diarrhea and abdominal pain. Serious encapsulated-bacterial infections, including meningococcal infection, require specific prevention and risk management. Hemolysis can occur if treatment is stopped.
Syfovre can cause eye discomfort, floaters and bleeding on the ocular surface. Serious risks include endophthalmitis, retinal detachment, retinal vascular inflammation and increased conversion to wet macular degeneration. Systemic and ophthalmic safety profiles belong to their respective products.
References and further reading
This reference draws on Superpower’s Peptide Library and the research linked below. Peptide Dossier’s editorial team adapted and integrated the material on .
- 10.1056/NEJMoa2029073Complement inhibition and PEGASUSCited in: Complement inhibition and PEGASUS
- pi.apellis.comProduct-specific FDA approvalsCited in: Product-specific FDA approvals
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Disclaimer: This article is for informational and educational purposes only. It is not intended as medical advice. The information presented is based on published research and should not be used as a substitute for professional medical guidance. Always consult a qualified healthcare provider before starting any peptide protocol. Some peptides discussed may not be approved for human use by the FDA or equivalent regulatory bodies.