Research statistics

Pegcetacoplan research: publications and trials

What has been published on Pegcetacoplan: PubMed publications, the kind of evidence they report and registered clinical trials. These are counts of research, not a judgment of whether Pegcetacoplan works or is safe. For what it is and what the studies found, see the Pegcetacoplan profile.

Automated weekly refresh · data through November 1, 2026

At a glance

Publications, all years

246

Since 1995

Publications in 2026 so far

Through November 1, 2026

Human clinical research

47.3%

107 of 226 classified

Registered clinical trials

ClinicalTrials.gov

Randomized controlled trials

Publications reporting an RCT in people

Systematic reviews and meta-analyses

Preclinical studies

5.8%

2 animal · 6 in vitro · 5 computational

Records that only mention it

127

Not counted above

Status in the library: FDA-approved. 20 publications are not yet classified by study type.

Publications by year

Pegcetacoplan publications by year and evidence

Counted PubMed publications by year of first publication. * 2026 runs to November 1, 2026. A larger bar is more publications, not stronger evidence.

Show the data

Year	Human clinical research	Preclinical (animal, in vitro, computational)	Reviews and meta-analyses	Other and not yet classified	Total
1995	0	1	0	0	1
2006	0	1	0	0	1
2008	0	1	0	0	1
2014	0	1	0	0	1
2018	0	1	0	0	1
2019	1	0	0	0	1
2020	1	0	0	0	1
2021	3	0	4	7	14
2022	9	3	15	4	31
2023	13	2	16	10	41
2024	24	1	9	17	51
2025	27	0	14	7	48
2026 (to date)	29	2	14	9	54

Study types

Review	65
Randomized controlled trial	33
Case report	32
Editorial, letter or other	28
Observational human study	25
Not yet classified	20
Other clinical trial	17
In vitro study	6
Computational study	5
Correction or retraction notice	5
Systematic review	4
Meta-analysis	3
Animal study	2
Guideline or consensus	1

Research areas

From MeSH major topics. A paper can be in several areas.

Immune system	116
Tissue repair	31
Metabolic health	26
Cardiovascular	16
Reproductive and sexual health	16
Musculoskeletal	8
Dermatology	7
Neurological and mental health	7
Oncology	7
Antimicrobial and infectious disease	6
Endocrinology	3
Cognitive health	2

Registered clinical trials

0 of 18 trials completed at least a year before the cutoff have neither posted results nor a linked results publication. A registration is not a result.

Completed: 19 · Recruiting: 5 · Active, not recruiting: 4 · Terminated: 3 · Not yet recruiting: 2 · Approved for marketing: 1 · Withdrawn: 1
Phase not applicable: 3 · Phase 1: 6 · Phase 1 / Phase 2: 2 · Phase 2: 10 · Phase 2 / Phase 3: 1 · Phase 3: 12 · Phase 4: 1

Trial	Status	Phase	Started	Results
To Assess the Efficacy and Safety of Pegcetacoplan for the Prevention of Recurrent C3G or Primary IC-MPGN in Kidney Transplant RecipientsNCT07898774 · 30 enrolled	Not yet recruiting	Phase 3	2027-01	—
A Sequential Phase 2/3 Study of APL2 in Patients With Focal Segmental GlomerulosclerosisNCT07213960 · 270 enrolled	Recruiting	Phase 2, Phase 3	2026-12	—
Safely Quenching Complement in Stroke SurvivorsNCT07495722 · 20 enrolled	Not yet recruiting	Phase 1	2026-10	—
Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3G or IC-MPG: A Multi-Country StudyNCT07746895 · 150 enrolled	Recruiting	Phase 4	2026-08	—
Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults at High Risk of Delayed Graft Function (DGF) Following Kidney Allograft TransplantationNCT07020832	Withdrawn	Phase 3	2026-05	—
Study to Evaluate a Pegcetacoplan (Syfovre®) Prefilled SyringeNCT07214740 · 44 enrolled	Completed	Phase 3	2025-10	—
Study to Assess the Adverse Events and How Intravitreal ABBV-6628 Moves Through the Body of Adult Participants With Geographic Atrophy Secondary to Age-Related Macular DegenerationNCT07160179 · 66 enrolled	Recruiting	Phase 1, Phase 2	2025-08	—
A Study to Investigate Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD), and Immunogenicity of RO7669330 in Participants With Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD)NCT06961370 · 27 enrolled	Terminated	Phase 1	2025-07	—
A Phase 2, Randomized, Placebo Controlled, Multicenter, Masked Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Multidose APL 3007 in Combination With Syfovre/Pegcetacoplan (APL-2) in Patients Diagnosed With Geographic Atrophy Secondary to Age Related Macular DegeneratioNCT07215390 · 240 enrolled	Recruiting	Phase 2	2025-06	—
A Study to Test Whether BI 771716 Helps People With an Advanced Form of Age-related Macular Degeneration (AMD) Called Geographic AtrophyNCT06722157 · 202 enrolled	Active, not recruiting	Phase 2	2025-01	—
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 DegenerationNCT06161584 · 300 enrolled	Active, not recruiting	Phase not applicable	2023-09	—
A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)NCT05776472 · 165 enrolled	Active, not recruiting	Phase not applicable	2023-06	—
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 GlomerulonephritisNCT05809531 · 100 enrolled	Active, not recruiting	Phase 3	2023-05	—
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)NCT05096403 · 24 enrolled	Completed	Phase 3	2022-10	Posted
A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell TransplantationNCT05148299 · 12 enrolled	Completed	Phase 2	2022-02	Posted
Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative GlomerulonephritisNCT05067127 · 124 enrolled	Completed	Phase 3	2021-11	Posted
Study Assessing the Safety and Efficacy of Pegcetacoplan in Post-Transplant Recurrence of C3G or IC-MPGNNCT04572854 · 13 enrolled	Completed	Phase 2	2021-09	Posted
An Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan (APL-2) in Subjects With Geographic Atrophy Secondary to AMDNCT04770545 · 792 enrolled	Completed	Phase 3	2021-03	Posted
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)NCT04901936 · 12 enrolled	Recruiting	Phase 2	2021-02	—
MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)NCT04579666 · 249 enrolled	Terminated	Phase 2	2020-09	Posted
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNHNCT04085601 · 53 enrolled	Completed	Phase 3	2019-08	Posted
Study to Evaluate the Safety of Intravitreal APL-2 in Patients Diagnosed With Geographic AtrophyNCT03777332 · 19 enrolled	Completed	Phase 1	2018-11	—
Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy With Sham Injections in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular DegenerationNCT03525600 · 621 enrolled	Completed	Phase 3	2018-08	Posted
A Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy With Sham Injections in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular DegenerationNCT03525613 · 637 enrolled	Completed	Phase 3	2018-08	Posted
Pegcetacoplan Long Term Safety and Efficacy Extension StudyNCT03531255 · 137 enrolled	Completed	Phase 3	2018-08	—
A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNHNCT03593200 · 4 enrolled	Completed	Phase 2	2018-08	Posted
Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)NCT03500549 · 80 enrolled	Completed	Phase 3	2018-06	Posted
Phase II Study Assessing Safety and Efficacy of APL-2 in GlomerulopathiesNCT03453619 · 21 enrolled	Completed	Phase 2	2018-02	Posted
Pegcetacoplan (APL-2) in Neovascular AMDNCT03465709 · 17 enrolled	Terminated	Phase 1, Phase 2	2018-02	Posted
Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)NCT03226678 · 24 enrolled	Completed	Phase 2	2017-08	Posted
Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.NCT02588833 · 23 enrolled	Completed	Phase 1	2015-12	Posted
Study of Pegcetacoplan (APL-2) Therapy in Patients With Geographic AtrophyNCT02503332 · 246 enrolled	Completed	Phase 2	2015-09	Posted
A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNHNCT02264639 · 9 enrolled	Completed	Phase 1	2015-02	Posted
Assessment of Safety, Tolerability and Pharmacokinetics of Intravitreal Pegcetacoplan (APL-2) for Patients With Wet AMDNCT02461771 · 13 enrolled	Completed	Phase 1	2015-01	Posted
C3G/Primary IC-MPGN EAPNCT04729062	Approved for marketing	Phase not applicable	—	—

Human trials and evidence reviews

Meta-analyses, systematic reviews and trials in people, newest first.

Efficacy and Safety of the C3 Inhibitor Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria: A Systematic Review and Meta-Analysis.
European journal of haematology · June 19, 2026 · Meta-analysis · cited 0 times · open access
PMID 42322093 · DOI 10.1111/ejh.70247 · Reviews and meta-analyses
Pegcetacoplan Delivers Real-World Therapeutic Benefits and Reduces Disease Burden for Patients With Paroxysmal Nocturnal Haemoglobinuria: A Systematic Literature Review of Pegcetacoplan Real-World Clinical and Patient-Reported Outcomes.
European journal of haematology · May 25, 2026 · Meta-analysis · cited 0 times · open access
PMID 42185224 · DOI 10.1111/ejh.70228 · Reviews and meta-analyses
Efficacy of Intravitreal Pegcetacoplan vs Avacincaptad Pegol in Patients With Geographic Atrophy.
Journal of vitreoretinal diseases · October 17, 2025 · Meta-analysis · cited 3 times · open access
PMID 41112493 · DOI 10.1177/24741264251379842 · Reviews and meta-analyses
Methodological pitfalls in indirect treatment comparisons: insights from a recent systematic review and analysis for C3 glomerulopathy.
Journal of comparative effectiveness research · August 9, 2026 · Systematic review · cited 0 times · open access
PMID 42561117 · DOI 10.57264/cer-2026-0054 · Reviews and meta-analyses
Efficacy and safety of complement inhibitors in patients with geographic atrophy associated with age-related macular degeneration: a network meta-analysis of randomized controlled trials.
Frontiers in pharmacology · November 12, 2024 · Systematic review · cited 4 times · open access
PMID 39600369 · DOI 10.3389/fphar.2024.1410172 · Reviews and meta-analyses
Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations.
Advances in therapy · April 18, 2023 · Systematic review · cited 22 times · open access
PMID 37072660 · DOI 10.1007/s12325-023-02510-4 · Reviews and meta-analyses
Pegcetacoplan in paroxysmal nocturnal hemoglobinuria: A systematic review on efficacy and safety.
Research and practice in thrombosis and haemostasis · July 1, 2022 · Systematic review · cited 14 times · open access
PMID 35949886 · DOI 10.1002/rth2.12781 · Reviews and meta-analyses
Indirect treatment comparisons find enhanced effectiveness of pegcetacoplan versus crovalimab in both complement inhibitor-naïve and -experienced patients with paroxysmal nocturnal hemoglobinuria.
Journal of comparative effectiveness research · August 24, 2026 · Randomized controlled trial · cited 0 times · open access
PMID 42636034 · DOI 10.57264/cer-2026-0015 · Human clinical research
Pegcetacoplan Versus Iptacopan for the Treatment of Patients with C3 Glomerulopathy: Indirect Treatment Comparisons.
Advances in therapy · May 15, 2026 · Randomized controlled trial · cited 0 times · open access
PMID 42138781 · DOI 10.1007/s12325-026-03614-3 · Human clinical research
Pegcetacoplan for Adolescents with C3 Glomerulopathy or Primary Immune Complex Membranoproliferative GN: Phase 3 VALIANT Subgroup Analysis.
Clinical journal of the American Society of Nephrology : CJASN · May 14, 2026 · Randomized controlled trial · cited 1 times · open access
PMID 42133432 · DOI 10.2215/cjn.0000001077 · Human clinical research

Most cited

By OpenAlex citation count. Citations measure attention, not quality or correctness.

Complement C3 Inhibitor Pegcetacoplan for Geographic Atrophy Secondary to Age-Related Macular Degeneration: A Randomized Phase 2 Trial.
Ophthalmology · July 16, 2019 · Randomized controlled trial · cited 525 times · open access
PMID 31474439 · DOI 10.1016/j.ophtha.2019.07.011 · Human clinical research
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.
Lancet (London, England) · October 1, 2023 · Randomized controlled trial · cited 520 times · open access
PMID 37865470 · DOI 10.1016/s0140-6736(23)01520-9 · Human clinical research
Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria.
The New England journal of medicine · March 1, 2021 · Randomized controlled trial · cited 440 times · open access
PMID 33730455 · DOI 10.1056/nejmoa2029073 · Human clinical research
Complement Factor D as a Strategic Target for Regulating the Alternative Complement Pathway.
Frontiers in immunology · September 9, 2021 · Review · cited 137 times · open access
PMID 34566967 · DOI 10.3389/fimmu.2021.712572 · Reviews and meta-analyses
C3 inhibition with pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria treated with eculizumab.
American journal of hematology · September 11, 2020 · Other clinical trial · cited 133 times · open access
PMID 33464651 · DOI 10.1002/ajh.25960 · Human clinical research

Latest publications

REAL-WORLD RETINAL VASCULITIS EVENTS REPORTED WITH FDA-APPROVED INTRAVITREAL ANTI-VEGF AND COMPLEMENT INHIBITOR AGENTS: An Analysis of FAERS Data.
Retina (Philadelphia, Pa.) · November 1, 2026 · Observational human study · cited 1 times
PMID 42627260 · DOI 10.1097/iae.0000000000004980 · Human clinical research
Retinitis and Choroiditis After the Seventh Intravitreal Pegcetacoplan Injection.
Journal of vitreoretinal diseases · October 26, 2026 · Case report · cited 0 times · open access
PMID 42947883 · DOI 10.1177/24741264261497612 · Human clinical research
Temporal, Phenotypic, and Treatment-Context Heterogeneity in Retinal Vasculitis Reporting with Pegcetacoplan: A United States Pharmacovigilance Study.
Journal of personalized medicine · October 16, 2026 · Observational human study · cited 0 times · open access
PMID 42930807 · DOI 10.3390/jpm16100533 · Human clinical research
A Systematic Review of Complement-Targeting Therapies for Geographic Atrophy: Insights into Advanced Drug Delivery Systems and Gene-Based Approaches.
Pharmaceutics · October 7, 2026 · Review · cited 0 times · open access
PMID 42942801 · DOI 10.3390/pharmaceutics18101269 · Reviews and meta-analyses
ONE-YEAR RESULTS OF GEOGRAPHIC ATROPHY PATIENTS WITH PREEXISTING nAMD TREATED WITH BOTH ANTI-VEGF THERAPY AND PEGCETACOPLAN.
Retina (Philadelphia, Pa.) · October 1, 2026 · Observational human study · cited 1 times
PMID 42467820 · DOI 10.1097/iae.0000000000004915 · Human clinical research
Podocyte and tubular vacuolization in patients receiving pegcetacoplan.
Kidney international · September 26, 2026 · Case report · cited 0 times
PMID 42800616 · DOI 10.1016/j.kint.2026.09.014 · Human clinical research
Distinct Pharmacodynamic Complement Biomarker Signatures Define Iptacopan and Pegcetacoplan Treatment in C3 Glomerulopathy.
Kidney international reports · September 18, 2026 · Observational human study · cited 0 times · open access
PMID 42898424 · DOI 10.1016/j.ekir.2026.107087 · Human clinical research
Breakthrough Hemolysis in Paroxysmal Nocturnal Hemoglobinuria: Mechanistic Insights and Management Strategies.
Transfusion · September 17, 2026 · Review · cited 0 times · open access
PMID 42755219 · DOI 10.1111/trf.70379 · Reviews and meta-analyses
Two-Year Experience With Intravitreal Pegcetacoplan for the Treatment of Geographic Atrophy in Clinical Practice.
American journal of ophthalmology · September 11, 2026 · Other clinical trial · cited 0 times
PMID 42727684 · DOI 10.1016/j.ajo.2026.09.011 · Human clinical research
Clinical improvement of acquired partial lipodystrophy in an adolescent with C3 glomerulopathy treated with pegcetacoplan.
Pediatric nephrology (Berlin, Germany) · September 3, 2026 · Case report · cited 0 times
PMID 42690456 · DOI 10.1007/s00467-026-07504-2 · Human clinical research

Browse all 246 publications in the dashboard

How this is counted

PubMed records matched by the names below, counted when the peptide is in the title, indexed by MeSH, or confirmed from the abstract. The exact query can be rerun on Europe PMC. Methodology

Search terms: APL-2; Aspaveli; Empaveli; Pegcetacoplan; Syfovre

((TITLE_ABS:"APL-2" OR CHEM:"APL-2" OR TITLE_ABS:"APL2" OR CHEM:"APL2") OR (TITLE_ABS:"Aspaveli" OR CHEM:"Aspaveli") OR (TITLE_ABS:"Empaveli" OR CHEM:"Empaveli") OR (TITLE_ABS:"Pegcetacoplan" OR CHEM:"Pegcetacoplan") OR (TITLE_ABS:"Syfovre" OR CHEM:"Syfovre")) AND SRC:MED

Cite this page

Peptide Library. (2026, November 2). Pegcetacoplan research statistics (Version weekly-2026-11-01) [Statistic]. Peptide Library. https://peptidelibrary.app/research/peptides/pegcetacoplan