Approved & therapeutic
Pegcetacoplan
Empaveli, Syfovre
Pegcetacoplan is a pegylated cyclic peptide complement C3 inhibitor, approved for paroxysmal nocturnal haemoglobinuria and, as an intravitreal formulation, for geographic atrophy secondary to age-related macular degeneration.
Also known as: Empaveli, Syfovre, APL-2
- Status
- Pegcetacoplan is approved by the U.S. FDA and has official prescribing information.
- Evidence
- Approved labeling. Official prescribing information exists, based on clinical trials in people.
- Half-life
- Approximately 8 days for the systemic formulation.
- How it's taken
- Subcutaneous infusion twice weekly, or intravitreal injection for the ophthalmic indication
- Mechanism
- Immune modulation
How Pegcetacoplan works
Pegcetacoplan binds complement C3 and C3b, blocking the complement cascade at the central convergence point of all three activation pathways. In paroxysmal nocturnal haemoglobinuria this prevents both intravascular haemolysis, which C5 inhibitors already address, and the extravascular haemolysis caused by C3 fragment deposition on red cells that C5 inhibitors do not.
What research has looked at
- In the PEGASUS trial it was superior to eculizumab for haemoglobin improvement in paroxysmal nocturnal haemoglobinuria, by additionally controlling extravascular haemolysis.
- Acting at C3 rather than C5 addresses a mechanism of ongoing anaemia that persists in many patients on C5 inhibitors.
- The intravitreal formulation reduced the rate of geographic atrophy lesion growth in age-related macular degeneration, the first treatment to do so.
Evidence level: Approved labeling. Official prescribing information exists, based on clinical trials in people. These are areas studied or reported, not promised effects.
Reported side effects and risks
- Boxed warning for serious infections caused by encapsulated bacteria. Blocking complement at C3 impairs defence against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae type b; vaccination is required before starting, and it is available only through a REMS programme.
- Complement inhibition at C3 is broader than at C5, so the infection risk is correspondingly broader.
- Injection-site reactions with the subcutaneous formulation, which requires infusion twice weekly via a pump.
- For the intravitreal formulation: increased incidence of exudative macular degeneration, and rare cases of retinal vasculitis.
- Discontinuation can precipitate severe haemolysis in paroxysmal nocturnal haemoglobinuria and requires monitoring.
Sources
- FDA approved-drug listing for pegcetacoplan · U.S. Food & Drug Administration
- DailyMed prescribing information for pegcetacoplan · NIH · DailyMed
- PubMed research on pegcetacoplan complement C3 PEGASUS · NIH · National Library of Medicine
- DrugBank record for pegcetacoplan · DrugBank
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Pegcetacoplan FAQ
What is Pegcetacoplan?
Pegcetacoplan is a pegylated cyclic peptide complement C3 inhibitor, approved for paroxysmal nocturnal haemoglobinuria and, as an intravitreal formulation, for geographic atrophy secondary to age-related macular degeneration.
How does Pegcetacoplan work?
Pegcetacoplan binds complement C3 and C3b, blocking the complement cascade at the central convergence point of all three activation pathways. In paroxysmal nocturnal haemoglobinuria this prevents both intravascular haemolysis, which C5 inhibitors already address, and the extravascular haemolysis caused by C3 fragment deposition on red cells that C5 inhibitors do not.
Is Pegcetacoplan FDA approved?
Yes. Pegcetacoplan is approved by the U.S. FDA and has official prescribing information. Evidence level: approved labeling. Official prescribing information exists, based on clinical trials in people.
What is the half-life of Pegcetacoplan?
Reported half-life: Approximately 8 days for the systemic formulation.
How is Pegcetacoplan taken?
Subcutaneous infusion twice weekly, or intravitreal injection for the ophthalmic indication.
What side effects have been reported with Pegcetacoplan?
Reported in the sources: Boxed warning for serious infections caused by encapsulated bacteria. Blocking complement at C3 impairs defence against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae type b; vaccination is required before starting, and it is available only through a REMS programme; Complement inhibition at C3 is broader than at C5, so the infection risk is correspondingly broader; Injection-site reactions with the subcutaneous formulation, which requires infusion twice weekly via a pump; For the intravitreal formulation: increased incidence of exudative macular degeneration, and rare cases of retinal vasculitis; Discontinuation can precipitate severe haemolysis in paroxysmal nocturnal haemoglobinuria and requires monitoring.
Educational summary compiled from the sources listed above. Not medical advice. Peptide Vault does not sell, source or recommend any compound. Talk to a qualified clinician before starting or changing anything.