Heart & blood Complement C3 inhibitor (PEG peptide)
Pegcetacoplan
Regulatory status
Status at a glance
503A compounding status
Not nominated
We found no pending nomination for the 503A bulks list. Unless the substance has another legal pathway (for example, a USP monograph or use in an FDA-approved drug), 503A pharmacies generally may not compound with it.
- FDA approval
- An FDA-approved product contains this peptide
- 503B outsourcing
- Not determined
- Anti-doping (WADA)
- Not on the WADA Prohibited List
- DEA schedule
- Not a controlled substance
About peptide status
Most peptides discussed here are not FDA-approved for any use, and products labeled “research use only” are not for human use. Removal from Category 2 does not authorize compounding. PCAC votes are non-binding recommendations; FDA and HHS make the final decisions.
This is not medical or legal advice, and we do not publish dosing, sourcing or reconstitution information. Talk with a licensed clinician before using any peptide.
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Key takeaway
Pegcetacoplan is a peptide that blocks complement protein C3, a central node of the immune system's complement cascade, and is sold as two distinct products: Empaveli for the blood disorder paroxysmal nocturnal hemoglobinuria and two rare kidney diseases, and Syfovre, an eye injection for geographic atrophy. By acting at C3, it controls complement-driven damage more broadly than drugs that block the later protein C5. Empaveli has been FDA-approved in the U.S. since 2021 and Syfovre since 2023.
At a glance
- What it is
- A pegylated peptide inhibitor of complement C3
- Approved for
- Paroxysmal nocturnal hemoglobinuria and two rare kidney diseases, C3 glomerulopathy and primary IC-MPGN (Empaveli), and geographic atrophy from age-related macular degeneration (Syfovre)
- Key fact
- In the PEGASUS trial, Empaveli was superior to the C5 inhibitor eculizumab for raising hemoglobin in PNH
- Route
- Subcutaneous infusion (Empaveli); intravitreal injection (Syfovre)
- Evidence strength
- FDA approved
Summary
Pegcetacoplan is an FDA-approved peptide that blocks complement C3. Learn how Empaveli and Syfovre work, what each is approved for, and key safety points.
What is pegcetacoplan?
Pegcetacoplan is a pegylated cyclic peptide that binds complement component C3 and its activation fragment C3b. The complement system is part of innate immunity, and C3 sits at the center of its cascade, so blocking C3 dampens complement activity broadly. The same molecule is marketed as two separate products for very different diseases: Empaveli, a subcutaneous treatment for paroxysmal nocturnal hemoglobinuria (PNH), and Syfovre, an eye injection for geographic atrophy. It was developed under the code APL-2, with Empaveli approved in 2021 and Syfovre in 2023.
How does pegcetacoplan work?
Pegcetacoplan binds C3, the protein at the hub of the complement cascade, blocking its activation and the downstream steps that lead to cell destruction. In PNH, red blood cells lack the surface proteins that normally protect them from complement, so they are destroyed both within blood vessels and, importantly, by the earlier C3 step on the cell surface. Because pegcetacoplan acts at C3, it controls both pathways of this destruction, which is broader than the older drugs that block only the later C5 protein.
In the eye, complement overactivity is thought to contribute to the slow retinal cell loss of geographic atrophy, and injecting pegcetacoplan into the eye is intended to slow that progression. In the PEGASUS trial in PNH, pegcetacoplan was superior to the C5 inhibitor eculizumab for improving hemoglobin over 16 weeks.
Is it FDA-approved?
Editor’s note
Status: Yes. The FDA has approved pegcetacoplan under two brand names, as the table shows: Year | Product | Approved for 2021 | Empaveli | PNH 2023 | Syfovre | Geographic atrophy secondary to age-related macular degeneration 2025 | Empaveli | C3 glomerulopathy and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN), 2 rare kidney diseases Empaveli carries a boxed warning for serious infections caused by encapsulated bacteria, so it requires vaccination against those bacteria and is available only through a restricted program.
Category: FDA-approved · Primary body area: Heart & blood
Yes. The FDA has approved pegcetacoplan under two brand names, as the table shows:
| Year | Product | Approved for |
|---|---|---|
| 2021 | Empaveli | PNH |
| 2023 | Syfovre | Geographic atrophy secondary to age-related macular degeneration |
| 2025 | Empaveli | C3 glomerulopathy and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN), 2 rare kidney diseases |
Empaveli carries a boxed warning for serious infections caused by encapsulated bacteria, so it requires vaccination against those bacteria and is available only through a restricted program.
What is pegcetacoplan prescribed for?
Empaveli is prescribed for adults with paroxysmal nocturnal hemoglobinuria, and for people 12 and older with C3 glomerulopathy or primary IC-MPGN to reduce protein in the urine. Syfovre is prescribed for geographic atrophy, an advanced form of dry age-related macular degeneration, given as an injection into the eye by an ophthalmologist.
What are the side effects?
According to the prescribing information, common side effects include:
- For Empaveli: injection-site reactions, infections, diarrhea, and abdominal pain
- For Syfovre: eye discomfort, floaters, and bleeding on the surface of the eye
More serious considerations include:
- For Empaveli: serious infections with encapsulated bacteria such as meningococcus, requiring vaccination and a risk management program, and the possibility of hemolysis if treatment is stopped
- For Syfovre: eye infection (endophthalmitis), retinal detachment, inflammation of retinal blood vessels, and a higher rate of converting to wet macular degeneration
Sources: FDA prescribing information for Empaveli and Syfovre; the PEGASUS trial (Hillmen et al., New England Journal of Medicine, 2021, DOI: 10.1056/NEJMoa2029073), indexed on PubMed. This document is educational and does not constitute medical advice.
Registered human trials
0 registered human trials on ClinicalTrials.gov that we reviewed
Sources
- [1]
- [2]
approved (PDF)
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