Peptide
Pegcetacoplan (Empaveli)
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.
- Updated
- How we grade
- 46 studies cited
Research notice. This page is for education and research. Peptides may not be FDA-approved for human use and may only be legally available for research. Talk to a qualified clinician before considering any peptide.
- Best evidence
- Grade A
- Conditions studied
- 1
- Outcomes
- 10
- Graded outcomes
- 5131
Evidence by condition
- Strong
- Moderate
- Limited
- Very limited
General
Key findings
- Hemoglobin Improvement vs EculizumabImproves (the measure goes up)
- Extravascular Hemolysis ControlImproves (the measure goes up)
- Safety ProfileImproves (the measure goes up)
Studies cited
46 studies from PubMed
- Safety and efficacy of pegcetacoplan treatment for cold agglutinin disease and warm antibody autoimmune hemolytic anemia.
- Efficacy and Safety of Pegcetacoplan in Kidney Transplant Recipients With Recurrent Complement 3 Glomerulopathy or Primary Immune Complex Membranoproliferative Glomerulonephritis.
- Ravulizumab and other complement inhibitors for the treatment of autoimmune disorders.
- Pegcetacoplan for the treatment of warm autoimmune hemolytic anemia: a case report.
- Trial of Pegcetacoplan in C3 Glomerulopathy and Immune-Complex MPGN.
- Efficacy, Safety, and Quality of Life of Pegcetacoplan in Japanese Patients with Paroxysmal Nocturnal Hemoglobinuria Treated within the Phase 3 PEGASUS Trial.
- Ocular Adverse Events Associated with Pegcetacoplan and Avacincaptad Pegol for Geographic Atrophy: A Population-Based Pharmacovigilance Study.
- Efficacy and Safety Maintained up to 3 Years in Adults with Paroxysmal Nocturnal Hemoglobinuria Receiving Pegcetacoplan.
- Sterile Intraocular Inflammation Following Intravitreal Injections: Pathogenesis, Clinical Features, and Management.
- Visual Function Benefit After Treatment With Pegcetacoplan: Microperimetry Analysis From the Phase 3 Oaks Trial.
- Breakthrough hemolysis in paroxysmal nocturnal hemoglobinuria throughout clinical trials: from definition to clinical practice.
- Treatment of geographic atrophy: an update on data related to pegcetacoplan.
- Pegcetacoplan Treatment and Consensus Features of Geographic Atrophy Over 24 Months.
- Autoimmune Hemolytic Anemias: Challenges in Diagnosis and Therapy.
- Safety and Efficacy of Pegcetacoplan in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria over 48 Weeks: 307 Open-Label Extension Study.
- Complement inhibitors in pediatric kidney diseases: new therapeutic opportunities.
- Severe Intraocular Inflammation Following Intravitreal Faricimab.
- Hemolysis events in the phase 3 PEGASUS study of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria.
- Pegcetacoplan: A New Opportunity for Complement Inhibition in PNH
- Pegcetacoplan (Syfovre) for geographic atrophy in age-related macular degeneration.
- Geographic atrophy: Mechanism of disease, pathophysiology, and role of the complement system.
- WITHDRAWN: Pegcetacoplan in the treatment of geographic atrophy.
- Pegcetacoplan for the treatment of geographic atrophy secondary to age-related macular degeneration (OAKS and DERBY): two multicentre, randomised, double-masked, sham-controlled, phase 3 trials.
- Advancements in the treatment of geographic atrophy: focus on pegcetacoplan in age-related macular degeneration.
- Clinical Safety and Efficacy of Pegcetacoplan in a Phase 2 Study of Patients with C3 Glomerulopathy and Other Complement-Mediated Glomerular Diseases.
- Targeting complement in IgA nephropathy.
- Pegcetacoplan for Treating Paroxysmal Nocturnal Haemoglobinuria: An Evidence Review Group Perspective of a NICE Single Technology Appraisal.
- Correction to: Pegcetacoplan: A Review in Paroxysmal Nocturnal Haemoglobinuria.
- Treatment of dry age-related macular degeneration: A review.
- Novel targeted C3 inhibitor pegcetacoplan for paroxysmal nocturnal hemoglobinuria
- Pegcetacoplan: A Review in Paroxysmal Nocturnal Haemoglobinuria
- Safety and efficacy of pegcetacoplan in paroxysmal nocturnal hemoglobinuria
- Association of Pegcetacoplan With Progression of Incomplete Retinal Pigment Epithelium and Outer Retinal Atrophy in Age-Related Macular Degeneration: A Post Hoc Analysis of the FILLY Randomized Clinical Trial.
- Pegcetacoplan treatment for geographic atrophy due to age-related macular degeneration: a plain language summary of the FILLY study.
- New Therapies for the Treatment of Warm Autoimmune Hemolytic Anemia.
- Safety and efficacy of pegcetacoplan in paroxysmal nocturnal hemoglobinuria.
- Pegcetacoplan in paroxysmal nocturnal hemoglobinuria: A systematic review on efficacy and safety.
- Pegcetacoplan versus eculizumab in patients with paroxysmal nocturnal haemoglobinuria (PEGASUS): 48-week follow-up of a randomised, open-label, phase 3, active-comparator, controlled trial.
- From discovery to approval: A brief history of the compstatin family of complement C3 inhibitors
- Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria
- Pegcetacoplan treatment in patients with paroxysmal nocturnal hemoglobinuria with suboptimal response to eculizumab: PRINCE study primary analysis
- Pegcetacoplan: First Approval
- Complement activation promoted by the lectin pathway mediates C3aR-dependent sarcoma progression and immunosuppression.
- Complement C3 Inhibitor Pegcetacoplan for Geographic Atrophy Secondary to Age-Related Macular Degeneration: A Randomized Phase 2 Trial.
- Complement and tissue factor-enriched neutrophil extracellular traps are key drivers in COVID-19 immunothrombosis.
- New Insights in Autoimmune Hemolytic Anemia: From Pathogenesis to Therapy Stage 1.