Pegcetacoplan
FDA approved · RxComplement C3 inhibitor
Also known as: Empaveli, Syfovre
Strong, human-grade evidence
- Regulatory standing. FDA-approved prescription medicine
- Human trials. Backed by controlled human studies
- Registered human trials. 35 registered studies on ClinicalTrials.gov, most advanced Phase 4
- 1 cited source. Includes primary / regulatory sources
Computed with the same four public criteria as every profile. The human-evidence grade on an indexed profile is set automatically and may change when it is hand-reviewed. It is not a safety score, not a purity rating, and not medical advice, and not a claim that this compound treats, cures, or prevents any condition.
What it is
Pegcetacoplan is a synthetic cyclic peptide attached to a polyethylene glycol (PEG) chain that binds complement protein C3 and its fragment C3b. By blocking C3, it is thought to dampen activation of the complement system, a part of the immune system that can damage red blood cells or retinal tissue in certain diseases.
What the evidence shows
It is FDA approved for paroxysmal nocturnal hemoglobinuria (as Empaveli, with a later expansion to certain kidney conditions) and, as the eye injection Syfovre, approved for geographic atrophy secondary to age-related macular degeneration. It has 34 registered human trials up to Phase 4 and over 300 published papers, while uses outside its approved indications remain under study.
Key facts
Registered human trials
Live from ClinicalTrials.gov, refreshed daily.
Pegcetacoplan (APL-2) in Neovascular AMD
Safely Quenching Complement in Stroke Survivors
Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3G or IC-MPG: A Multi-Country Study
Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Published literature
Live from Europe PMC. This is how much has been published, not how much is proven in humans.
Complement C3 Inhibitor Pegcetacoplan for Geographic Atrophy Secondary to Age-Related Macular Degeneration: A Randomized Phase 2 Trial.
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.
Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria.
Clinical Safety and Efficacy of Pegcetacoplan in a Phase 2 Study of Patients with C3 Glomerulopathy and Other Complement-Mediated Glomerular Diseases.
Sources
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