Peptide Association

Insights

NewsBlog
Start free intake

DisclosurePeptide Association LLC is a for-profit company. We receive compensation from the clinical providers listed on this site. We are not an accrediting body, a licensing board, or a government-affiliated organization. See exactly how we make money.

All peptides

Hematology · FDA Approved

Pegcetacoplan (Empaveli)

FDA-approved

How to ask your doctor
Written by Peptide Association Editorial TeamLast reviewed Editorial policy

01 · Overview

A pegylated cyclic peptide that inhibits complement component C3 and C3b, acting upstream of C5 in the complement cascade.

Because C5 inhibitors control intravascular hemolysis but not extravascular hemolysis in paroxysmal nocturnal hemoglobinuria (PNH), targeting C3 aims to control both (PMID 33730455).

02 · Research summary & key findings

FDA-approved in 2021 (Empaveli) for adults with PNH. In the PEGASUS trial in patients still anemic on eculizumab, pegcetacoplan was superior for hemoglobin change at week 16 (adjusted difference 3.84 g/dL), and 85% vs. 15% no longer needed transfusions; injection site reactions and diarrhea were more common (Hillmen et al., NEJM, 2021) (PMID 33730455). In the phase 3 VALIANT trial in C3 glomerulopathy or primary immune-complex MPGN, pegcetacoplan reduced proteinuria by about 68% relative to placebo (PMID 41337715), and the FDA extended its approval to these kidney diseases in 2025. It is given by subcutaneous infusion (PMID 33730455).

03 · Regulatory status

Pegcetacoplan (Empaveli) has received FDA approval and is available for clinical use under appropriate medical supervision. Consult a qualified healthcare provider for prescribing information.

Pegcetacoplan (Empaveli) is an FDA-approved medicine prescribed for the conditions on its label. It is not part of our intake, so we do not propose protocols for it. If you think it may be relevant to you, talk to your own doctor. How to ask your doctor.

04 · Administration routes

  • Subcutaneous infusion

References

  1. [1]Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria N Engl J Med, 2021. PMID 33730455
  2. [2]Trial of Pegcetacoplan in C3 Glomerulopathy and Immune-Complex MPGN N Engl J Med, 2025. PMID 41337715

Primary literature

Related peptides in Hematology

Disclaimer: This information is provided for educational and research purposes only. It is not intended as medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before starting any peptide therapy. The Peptide Association does not endorse or recommend any specific treatment protocol. See our editorial policy for how this content is produced and reviewed. Clinicians: join the network.