Pegcetacoplan
- Generic Name
- Pegcetacoplan
- Brand Names
- Empaveli, Syfovre, Aspaveli
- Drug Type
- Biotech
- CAS Number
- 2019171-69-6
- Unique Ingredient Identifier
- TO3JYR3BOU
- Background
Pegcetacoplan is a complement inhibitor indicated in the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Prior to its FDA approval, patients with PNH were typically treated with the C5 inhibiting monoclonal antibody eculizumab. Patients given eculizumab experienced less hemolysis caused by the membrane attack complex, but were still somewhat susceptible to hemolysis caused by C3b opsonization. Pegcetacoplan was developed out of a need for an inhibitor of complement mediated hemolysis further upstream of C5. Pegcetacoplan is a pegylated C3 inhibitor that can disrupt the processes leading to both forms of hemolysis that threaten patients with PNH.
Pegcetacoplan for subcutaneous use was granted FDA approval on 14 May 2021. In February 2023, pegcetacoplan for intravitreal use was approved by the FDA for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration.
- Indication
Pegcetacoplan is indicated to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). It is also indicated to treat geographic atrophy (GA) secondary to age-related macular degeneration.
- Associated Conditions
- Geographic Atrophy Secondary to Age-related Macular Degeneration, Paroxysmal Nocturnal Haemoglobinuria (PNH)
A Study to Test Whether BI 771716 Helps People With an Advanced Form of Age-related Macular Degeneration (AMD) Called Geographic Atrophy
- Conditions
- Macular Degeneration, Age-relatedGeographic Atrophy
- Interventions
- First Posted Date
- 2024-12-09
- Last Posted Date
- 2025-05-21
- Lead Sponsor
- Boehringer Ingelheim
- Target Recruit Count
- 180
- Registration Number
- NCT06722157
- Locations
- 🇺🇸
Associated Retina Consultants, Gilbert, Arizona, United States
🇺🇸Associated Retina Consultants, Ltd., Phoenix, Arizona, United States
🇺🇸Phoenix Retina Associates, Phoenix, Arizona, United States
A Prospective, Multicenter, Open-Label, Observational Phase 4 Study to Evaluate Real-World Safety, Tolerability, and Treatment Patterns of Pegcetacoplan (Syfovre) in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration
- First Posted Date
- 2023-12-08
- Last Posted Date
- 2025-02-25
- Lead Sponsor
- Apellis Pharmaceuticals, Inc.
- Target Recruit Count
- 300
- Registration Number
- NCT06161584
- Locations
- 🇺🇸
Illinois Retina Associates (01-037), Oak Park, Illinois, United States
🇺🇸California Retina Consultants (01-026), Bakersfield, California, United States
🇺🇸Retina-Vitreous Associates Medical Group (01-020), Beverly Hills, California, United States
An Open-Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Participants With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
- Conditions
- C3 GlomerulopathyDDDMembranoproliferative GlomerulonephritisComplement 3 GlomerulopathyMembranoproliferative Glomerulonephritis (MPGN)IC-MPGNC3 GlomerulonephritisComplement 3 GlomerulonephritisDense Deposit DiseaseImmune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)
- Interventions
- First Posted Date
- 2023-04-12
- Last Posted Date
- 2025-03-28
- Lead Sponsor
- Apellis Pharmaceuticals, Inc.
- Target Recruit Count
- 100
- Registration Number
- NCT05809531
- Locations
- 🇺🇸
Academic Medical Research Institute (01034), Los Angeles, California, United States
🇺🇸Children's Hospital Colorado (01037), Aurora, Colorado, United States
🇺🇸University of Florida, Department of Pediatric Nephrology (01010), Gainesville, Florida, United States
A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- First Posted Date
- 2023-03-20
- Last Posted Date
- 2025-05-14
- Lead Sponsor
- Swedish Orphan Biovitrum
- Target Recruit Count
- 200
- Registration Number
- NCT05776472
- Locations
- 🇦🇺
Liverpool Hospital, Liverpool, New South Wales, Australia
🇦🇺Calvary Mater Newcastle, Waratah, New South Wales, Australia
🇦🇺Sunshine Coast University Hospital, Birtinya, Queensland, Australia
Evaluate the PK Efficacy Safety and Tolerability of Pegcetacoplan in Patients With Thrombotic Microangiopathy
- Conditions
- Transplant-Associated Thrombotic Microangiopathy
- Interventions
- First Posted Date
- 2021-12-08
- Last Posted Date
- 2024-12-19
- Lead Sponsor
- Swedish Orphan Biovitrum
- Target Recruit Count
- 12
- Registration Number
- NCT05148299
- Locations
- 🇬🇷
University General Hospital "Attikon", Athens, Chaidari, Greece
🇫🇷CHU de Saint-Etienne, Saint-Priest-en-Jarez, France
🇬🇷General Hospital of Thessaloniki "G. Papanikolaou", Hematology Department - BMT Unit, Thessaloníki, Greece
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)
- First Posted Date
- 2021-10-27
- Last Posted Date
- 2024-04-11
- Lead Sponsor
- Swedish Orphan Biovitrum
- Target Recruit Count
- 57
- Registration Number
- NCT05096403
- Locations
- 🇬🇪
Ltd M. Zodelava Hematology Centre, Tbilisi, Georgia
🇬🇧Churchill Hospital, Oxford, United Kingdom
🇧🇪CHU de Liège, Liège, Belgium
Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
- Conditions
- C3GMembranoproliferative Glomerulonephritis (MPGN)IC-MPGNComplement 3 GlomerulopathyComplement 3 GlomerulonephritisDense Deposit DiseaseDDDC3 GlomerulopathyComplement 3 Glomerulopathy (C3G)C3 Glomerulonephritis
- Interventions
- Other: Placebo
- First Posted Date
- 2021-10-05
- Last Posted Date
- 2025-03-13
- Lead Sponsor
- Apellis Pharmaceuticals, Inc.
- Target Recruit Count
- 124
- Registration Number
- NCT05067127
- Locations
- 🇳🇱
University Medical Center Groningen, Groningen, Netherlands
🇳🇱Emma Kinderziekenhuis, Amsterdam UMC, Amsterdam, Netherlands
🇳🇱Radboud University Medical Center, Nijmegen, Netherlands
APL-2 and Pembrolizumab Versus APL-2, Pembrolizumab and Bevacizumab Versus Bevacizumab Alone for the Treatment of Recurrent Ovarian, Fallopian Tube, or Primary Peritoneal Cancer and Malignant Effusion
- Conditions
- Fallopian Tube Serous AdenocarcinomaOvarian Clear Cell AdenocarcinomaPrimary Peritoneal Endometrioid AdenocarcinomaFallopian Tube CarcinosarcomaOvarian Endometrioid AdenocarcinomaOvarian Serous AdenocarcinomaPrimary Peritoneal CarcinosarcomaRecurrent Ovarian CarcinomaFallopian Tube Clear Cell AdenocarcinomaOvarian Carcinosarcoma
- Interventions
- Procedure: BiopsyProcedure: Biospecimen CollectionOther: Questionnaire Administration
- First Posted Date
- 2021-06-09
- Last Posted Date
- 2025-05-11
- Lead Sponsor
- Roswell Park Cancer Institute
- Target Recruit Count
- 60
- Registration Number
- NCT04919629
- Locations
- 🇺🇸
Roswell Park Cancer Institute, Buffalo, New York, United States
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Hemoglobinuria
- Interventions
- First Posted Date
- 2021-05-26
- Last Posted Date
- 2022-07-20
- Lead Sponsor
- Apellis Pharmaceuticals, Inc.
- Target Recruit Count
- 12
- Registration Number
- NCT04901936
- Locations
- 🇹🇭
Phramongkutklao Hospital and College of Medicine, Bangkok, Thailand
🇨🇿Motol University Hospital, Prague, Czechia
🇺🇸Children's Hospital of Atlanta, Atlanta, Georgia, United States
An Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan (APL-2) in Subjects With Geographic Atrophy Secondary to AMD
- Conditions
- Geographic Atrophy Secondary to Age-related Macular Degeneration
- Interventions
- First Posted Date
- 2021-02-25
- Last Posted Date
- 2025-03-06
- Lead Sponsor
- Apellis Pharmaceuticals, Inc.
- Target Recruit Count
- 1200
- Registration Number
- NCT04770545
- Locations
- 🇺🇸
Northwestern Feinberg School of Medicine, Chicago, Illinois, United States
🇺🇸The Ohio State University, Columbus, Ohio, United States
🇺🇸Ophthalmic Consultants of Boston, Boston, Massachusetts, United States
Drug Development Updates
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