Pegcetacoplan
Also referenced as: Empaveli, Syfovre
A PEGylated cyclic peptide inhibitor of complement C3 with distinct systemic and ocular products.
- Evidence grade
- AStrong Clinical Evidence
Strong Clinical Evidence
- Regulatory status
- FDA Approved for Specific Indication
Empaveli is FDA-approved for adult PNH and specified C3G or primary IC-MPGN from age 12. Syfovre separately treats geographic atrophy secondary to AMD; product-specific criteria apply.
- Human data
- Yes
Registered clinical trials exist
- Last reviewed
- October 27, 2026
Overview
A PEGylated cyclic peptide inhibitor of complement C3 with distinct systemic and ocular products.
Empaveli is FDA-approved for adult PNH and specified C3G or primary IC-MPGN from age 12. Syfovre separately treats geographic atrophy secondary to AMD; product-specific criteria apply.
Empaveli and Syfovre have different routes, indications and safety profiles and must not be treated as interchangeable.
Why It's Being Studied
- PNH
- Complement kidney disease
- Geographic atrophy
Mechanism of Action
C3 and C3b inhibition reduces complement-pathway activity; formulation determines tissue exposure.
C3 and C3b inhibition reduces complement-pathway activity; formulation determines tissue exposure. Empaveli and Syfovre have different routes, indications and safety profiles and must not be treated as interchangeable.
From molecular target to clinical evidence
Tap each stage to see what happens.
Background: Clinical Peptide Medicines
Clinical peptide medicines include endocrine treatments, antimicrobial drugs, receptor blockers and targeted diagnostic or cancer agents. They do not share one mechanism or one set of indications.
An approval belongs to a specific medicine and indication. A diagnostic tracer, an antibiotic and a hormone are not interchangeable wellness products; read each profile's own mechanism and prescribing source.
Research Areas & Routes Studied
- PNH
- Complement kidney disease
- Geographic atrophy
- Subcutaneous approved systemic formulation
- Intravitreal approved ocular formulation
Evidence Summary
Disease-specific randomized evidence supports systemic complement-disease and ocular geographic-atrophy indications.
Higher tiers carry more weight. Filled tiers have evidence for Pegcetacoplan; tap to read it.
- Disease-specific randomized evidence supports systemic complement-disease and ocular geographic-atrophy indications.
2 cited sources.
- Prescribing label2
Human clinical evidence
- Disease-specific randomized evidence supports systemic complement-disease and ocular geographic-atrophy indications.
Reported Adverse Events in Research
- Systemic use carries serious encapsulated-bacterial infection risks and REMS requirements; ocular risks include endophthalmitis and retinal vasculitis or occlusion.
Limitations of Current Evidence
- Empaveli and Syfovre have different routes, indications and safety profiles and must not be treated as interchangeable.
- Findings apply to the product, route and population studied; they do not establish benefits or safety for unrelated wellness uses or research-vial products.
How to Read This Research
- Is this a single molecule or a family of medicines?
- Is the use diagnostic, therapeutic or investigational?
- Does the quoted outcome match the exact product, formulation and population?
- Use the compound-specific mechanism above; this diagram describes drug development, not a shared biological pathway.
- Historical approval, present availability and off-label use are distinct questions.
- For radiopharmaceuticals and hospital medicines, specialist handling is part of the treatment context.
- Indication
- The condition and population for which a medicine is approved.
- Antagonist
- A compound that blocks activity at a receptor.
- Radiopharmaceutical
- A medicine containing a radioactive isotope for imaging or treatment.
Compare With Other Compounds
| Pegcetacoplan | Trofinetide | |
|---|---|---|
| Evidence grade | A — Strong Clinical Evidence | B — Moderate Human Evidence |
| Regulatory status | FDA Approved for Specific Indication | FDA Approved for Specific Indication |
| Human data | Yes | Yes |
| Clinical trials | Registered trials exist | Registered trials exist |
| Research routes | Subcutaneous approved systemic formulation, Intravitreal approved ocular formulation | Oral approved medicine |
| Cited sources | 2 | 2 |
Frequently Asked Questions
Research References
- Prescribing label
Pegcetacoplan — prescribing label
- Prescribing label
SYFOVRE prescribing sources