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Pegcetacoplan (Empaveli)

Peptide

Pegcetacoplan is a PEGylated 15-amino acid cyclic peptide that inhibits complement C3. FDA-approved May 2021 for adults with paroxysmal nocturnal hemoglobinuria (PNH). First C3-targeted PNH therapy. Phase III PEGASUS trial: Superior to eculizumab for hemoglobin improvement (p<0.001); 85% vs 15% transfusion-free at 16 weeks. Inhibits both intravascular and extravascular hemolysis. Derived from compstatin family of C3 inhibitors. SC infusion 1080 mg twice weekly.

Quick Answer

What it is

Pegcetacoplan is a PEGylated 15-amino acid cyclic peptide that inhibits complement C3. FDA-approved May 2021 for adults with paroxysmal nocturnal hemoglobinuria (PNH).

Key findings

  • Grade A: Hemoglobin Improvement vs Eculizumab (Blood Health)
  • Grade A: Transfusion Independence (Blood Health)
  • Grade A: Extravascular Hemolysis Control (Blood Health)

Safety

No specific caution or interaction language was detected in the current summary/outcome notes.

⚠️ Research Notice

This peptide information is for educational and research purposes only. Peptides may not be FDA-approved for human use and may only be legally available for research purposes. Consult qualified healthcare professionals before considering any peptide compounds.

ℹ️ Quick Facts: Pegcetacoplan (Empaveli)

Quick Facts: Pegcetacoplan (Empaveli)

  • Best Evidence:Grade A
  • Conditions Studied:1
  • Research Outcomes:10
  • Grade A Findings:5
  • Grade B Findings:1
  • Key Effect:Blood Health
A5
B1
C3
D1
1 conditions · 10 outcomes

Detailed Outcomes

A
Hemoglobin Improvement vs Eculizumab
PEGASUS Phase III trial: Pegcetacoplan significantly superior to eculizumab for hemoglobin improvement (p<0.001). First therapy to demonstrate superiority over standard C5 inhibitor treatment in PNH.
large↑Improves
A
Transfusion Independence
PEGASUS trial: 85% of pegcetacoplan patients transfusion-free at 16 weeks vs 15% on eculizumab (p<0.001). Dramatic reduction in transfusion requirements.
large↑Improves
A
Extravascular Hemolysis Control
Unique mechanism: C3 inhibition prevents C3b opsonization, controlling extravascular hemolysis that C5 inhibitors cannot address. Addresses key limitation of prior PNH treatments.
large↑Improves
A
Complement-Inhibitor-Naive PNH
PRINCE Phase III trial: Superior to supportive care in treatment-naive PNH patients. Achieved hemoglobin stabilization and LDH reduction. Approved for both naive and C5i-experienced patients.
large↑Improves
A
Safety Profile
Well-tolerated in clinical trials. Most common AEs: injection site reactions, infections. Requires vaccination against encapsulated bacteria (meningococcal, pneumococcal). Self-administered subcutaneously.
moderate↑Improves
B
Muscle Mass/Function
18 human trials support this finding. Human clinical trial data available.
moderate↑Improves
C
Immune Function
11 preclinical studies support this finding. Primarily preclinical evidence.
moderate↑Improves
C
Kidney Function
3 preclinical studies support this finding. Primarily preclinical evidence.
small↑Improves
C
Corneal/Ocular Health
3 preclinical studies support this finding. Primarily preclinical evidence.
small↑Improves
D
Anti-Inflammatory Activity
2 preclinical studies support this finding. Primarily preclinical evidence.
small↑Improves

Research Citations (46)

Safety and efficacy of pegcetacoplan treatment for cold agglutinin disease and warm antibody autoimmune hemolytic anemia.
(2025)
PMID: 39486046
Efficacy and Safety of Pegcetacoplan in Kidney Transplant Recipients With Recurrent Complement 3 Glomerulopathy or Primary Immune Complex Membranoproliferative Glomerulonephritis.
(2025)
PMID: 39810766
Ravulizumab and other complement inhibitors for the treatment of autoimmune disorders.
(2025)
PMID: 39983521
Pegcetacoplan for the treatment of warm autoimmune hemolytic anemia: a case report.
(2025)
PMID: 40450956
Trial of Pegcetacoplan in C3 Glomerulopathy and Immune-Complex MPGN.
(2025)
PMID: 41337715
Efficacy, Safety, and Quality of Life of Pegcetacoplan in Japanese Patients with Paroxysmal Nocturnal Hemoglobinuria Treated within the Phase 3 PEGASUS Trial.
(2025)
PMID: 38615657
Ocular Adverse Events Associated with Pegcetacoplan and Avacincaptad Pegol for Geographic Atrophy: A Population-Based Pharmacovigilance Study.
(2025)
PMID: 40288597
Efficacy and Safety Maintained up to 3 Years in Adults with Paroxysmal Nocturnal Hemoglobinuria Receiving Pegcetacoplan.
(2025)
PMID: 40720060
Sterile Intraocular Inflammation Following Intravitreal Injections: Pathogenesis, Clinical Features, and Management.
(2025)
PMID: 40601512
Visual Function Benefit After Treatment With Pegcetacoplan: Microperimetry Analysis From the Phase 3 Oaks Trial.
(2025)
PMID: 39954920

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Gramicidin

Peptide

1 shared condition · 8 outcomes

Gramicidin is a cyclic peptide antibiotic from Bacillus brevis, first isolated 1941, FDA-approved 1955 as Neosporin component. Topical use only (lozenges, eye drops, ointments) due to hemolysis. Gramicidin D (mixture of A/B/C) used clinically. Ophthalmic study (n=91): Effective and safe for bacterial corneal ulceration. Combined with polymyxin B and neomycin. One of first membrane-active peptide antibiotics. SAFETY CONCERN: Highly hemolytic - systemic use contraindicated.