Pegcetacoplan
Also known as: Empaveli, Syfovre, APL-2, Aspaveli
A pegylated cyclic peptide C3 inhibitor, FDA-approved as Empaveli (2021, PNH; 2025, C3G/IC-MPGN) and Syfovre (2023, geographic atrophy).
Last updated
Mechanism of Action
Pegcetacoplan is a pair of synthetic cyclic peptides (from the compstatin family) joined to a polyethylene glycol chain. It binds complement component C3 and its fragment C3b, blocking C3 cleavage and downstream complement activation. Acting at C3 controls both the C3b coating that drives extravascular hemolysis in PNH and the terminal membrane-attack pathway, and the same mechanism is used in the retina to slow geographic atrophy.
- Half-life
- ~8 days (subcutaneous)
- Administration Routes
- Subcutaneous, Intravitreal
- Research Status
- Clinical
Reported Benefits
- Paroxysmal nocturnal hemoglobinuria (Empaveli): improved hemoglobin vs eculizumab
- C3 glomerulopathy and primary IC-MPGN (Empaveli): reduced proteinuria
- Geographic atrophy from AMD (Syfovre, intravitreal): slowed lesion growth
Potential Side Effects
- Boxed warning (Empaveli): serious infections from encapsulated bacteria
- Injection-site reactions, diarrhea, viral infections (Empaveli)
- Syfovre: endophthalmitis, retinal vasculitis, conversion to wet AMD
- Eye pain and floaters after intravitreal injection
Common Dosing
Empaveli is a subcutaneous infusion given twice weekly at a fixed dose, with required vaccination against encapsulated bacteria and enrollment in a safety program. Syfovre is a separate product injected into the eye by a retina specialist every 25 to 60 days. Dosing is set and supervised by the prescribing clinician.
The dosing information above is aggregated from research literature and anecdotal community reports for educational purposes only. It is not a recommendation, prescription, or medical advice. Peptides are sold as research chemicals only and are not intended for human use.
Contraindications
- Unresolved serious infection with encapsulated bacteria (Empaveli)
- Not vaccinated against encapsulated bacteria unless risk outweighed (Empaveli)
- Active eye or periocular infection, or active intraocular inflammation (Syfovre)
- Hypersensitivity to pegcetacoplan
No documented stacking partners for Pegcetacoplan yet.
- View
Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria
2021
- View
Trial of Pegcetacoplan in C3 Glomerulopathy and Immune-Complex MPGN
2025
- View
Pegcetacoplan for the treatment of geographic atrophy secondary to age-related macular degeneration (OAKS and DERBY): two multicentre, randomised, double-masked, sham-controlled, phase 3 trials
2023