At a glance
FDA approval FDA-approved 2 applications
Randomized controlled trials 32 of 337 publications on PubMed
Registered clinical trials 38 16 active
Adverse event reports 1,783 1,182 classified as serious
- Pegcetacoplan is an FDA-approved drug, marketed as Empaveli, Syfovre (first approval: May 14, 2021).
- PubMed lists 337 publications mentioning it, including 32 randomized controlled trials and 1 animal-only study.
- ClinicalTrials.gov lists 38 registered studies testing it (16 active).
- FDA's adverse event system (FAERS) holds 1,783 reports mentioning it; 1,182 classified as serious. Reports do not prove causation.
Research topics
Most frequent MeSH subject headings in the latest PubMed records (English, as indexed).
Regulatory status
Drugs@FDA applications
| Application | Product | Status | First approval | Sponsor |
| NDA215014 | EMPAVELI | Approved, marketed | May 14, 2021 | APELLIS PHARMS |
| NDA217171 | SYFOVRE | Approved, marketed | February 17, 2023 | APELLIS PHARMS |
Source: Drugs@FDA (openFDA) · verified October 30, 2026 · API query
FDA compounding safety list
Not on FDA's list of bulk drug substances that may present significant safety risks in compounding.
Source: FDA.gov — Category 2 bulk drug substances · verified October 30, 2026
Removal of a substance from a regulatory list does not mean it has been approved. A "research use only" label does not make a product safe or legal for human use.
Scientific publications
- All publications 337
- Human (MeSH) 199
- Animal only 1
- Clinical trials 40
- Randomized controlled trials 32
- Systematic reviews & meta-analyses 7
- Case reports 28
- Retracted 0
Counts are PubMed records that mention the peptide in the title or abstract. Categories overlap, and a mention is not the same as testing the peptide as a treatment. Each number opens the exact PubMed search. Effects seen in laboratory or animal studies may not occur in humans. A registered clinical trial does not show that a substance is effective or safe.
Source: PubMed (NCBI) · verified October 30, 2026
Clinical trials
- Registered
- 38
- Active
- 16
- With posted results
- 16
| Study | Status | Phase | Start |
| Pilot STUDY to Monitor Ex-vivo the Biological Effects of the C3 Inhibitor, PEGCETACOPLAN, on Activation of Complement in Fluid Phase and on Podocytes in Patients With C3G and Primary IC-MPGN NCT07904338 Mario Negri Institute for Pharmacological Research | Not yet recruiting | — | Mar 2027 |
| To Assess the Efficacy and Safety of Pegcetacoplan for the Prevention of Recurrent C3G or Primary IC-MPGN in Kidney Transplant Recipients NCT07898774 Swedish Orphan Biovitrum | Not yet recruiting | Phase 3 | January 21, 2027 |
| Safely Quenching Complement in Stroke Survivors NCT07495722 Columbia University | Not yet recruiting | Phase 1 | Oct 2026 |
| Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3G or IC-MPG: A Multi-Country Study NCT07746895 Swedish Orphan Biovitrum | Recruiting | Phase 4 | August 5, 2026 |
| Lesion-tailored, Fundus-controlled Perimetry in Geographic Atrophy (GA). NCT07582042 University of Utah | Recruiting | — | June 15, 2026 |
| Pegcetacoplan in Combination With Modified FOLFIRINOX for the Treatment of Metastatic Pancreatic Ductal Adenocarcinoma NCT07214298 Roswell Park Cancer Institute | Recruiting | Phase 1, Phase 2 | February 16, 2026 |
| 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 Degeneration NCT07160179 AbbVie | Recruiting | Phase 1, Phase 2 | August 13, 2025 |
| 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 Degeneratio NCT07215390 Apellis Pharmaceuticals, Inc. | Recruiting | Phase 2 | June 23, 2025 |
| A Study to Test Whether BI 771716 Helps People With an Advanced Form of Age-related Macular Degeneration (AMD) Called Geographic Atrophy NCT06722157 Boehringer Ingelheim | Active, not recruiting | Phase 2 | January 6, 2025 |
| Home Reported Outcomes in PNH NCT06411626 Novartis Pharmaceuticals | Recruiting | — | June 11, 2024 |
Show 28 more studies
| Study | Status | Phase | Start |
| 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 NCT06161584 Apellis Pharmaceuticals, Inc. | Active, not recruiting | — | September 28, 2023 |
| A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease NCT06092034 Rocket Pharmaceuticals Inc. | Recruiting | Phase 2 | September 5, 2023 |
| A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT05776472 Swedish Orphan Biovitrum | Active, not recruiting | — | June 26, 2023 |
| 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 NCT05809531 Apellis Pharmaceuticals, Inc. | Active, not recruiting | Phase 3 | May 29, 2023 |
| 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 NCT04919629 Roswell Park Cancer Institute | Recruiting | Phase 2 | April 27, 2023 |
| A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT04901936 Apellis Pharmaceuticals, Inc. | Recruiting | Phase 2 | February 4, 2021 |
| Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults at High Risk of Delayed Graft Function (DGF) Following Kidney Allograft Transplantation NCT07020832 Apellis Pharmaceuticals, Inc. | Withdrawn | Phase 3 | May 18, 2026 |
| Study to Evaluate a Pegcetacoplan (Syfovre®) Prefilled Syringe NCT07214740 Apellis Pharmaceuticals, Inc. | Completed | Phase 3 | October 24, 2025 |
| 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 Hoffmann-La Roche | Terminated | Phase 1 | July 16, 2025 |
| A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD) NCT05096403 Swedish Orphan Biovitrum Results posted | Completed | Phase 3 | October 20, 2022 |
| A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation NCT05148299 Swedish Orphan Biovitrum Results posted | Completed | Phase 2 | February 1, 2022 |
| Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis NCT05067127 Apellis Pharmaceuticals, Inc. Results posted | Completed | Phase 3 | November 12, 2021 |
| Study Assessing the Safety and Efficacy of Pegcetacoplan in Post-Transplant Recurrence of C3G or IC-MPGN NCT04572854 Apellis Pharmaceuticals, Inc. Results posted | Completed | Phase 2 | September 7, 2021 |
| An Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan (APL-2) in Subjects With Geographic Atrophy Secondary to AMD NCT04770545 Apellis Pharmaceuticals, Inc. Results posted | Completed | Phase 3 | March 4, 2021 |
| MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS) NCT04579666 Apellis Pharmaceuticals, Inc. Results posted | Terminated | Phase 2 | September 30, 2020 |
| A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH NCT04085601 Apellis Pharmaceuticals, Inc. Results posted | Completed | Phase 3 | August 27, 2019 |
| Study to Evaluate the Safety of Intravitreal APL-2 in Patients Diagnosed With Geographic Atrophy NCT03777332 Apellis Pharmaceuticals, Inc. | Completed | Phase 1 | November 5, 2018 |
| 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 Degeneration NCT03525600 Apellis Pharmaceuticals, Inc. Results posted | Completed | Phase 3 | August 31, 2018 |
| 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 Degeneration NCT03525613 Apellis Pharmaceuticals, Inc. Results posted | Completed | Phase 3 | August 31, 2018 |
| Pegcetacoplan Long Term Safety and Efficacy Extension Study NCT03531255 Apellis Pharmaceuticals, Inc. | Completed | Phase 3 | August 27, 2018 |
| A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH NCT03593200 Apellis Pharmaceuticals, Inc. Results posted | Completed | Phase 2 | August 16, 2018 |
| Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT03500549 Apellis Pharmaceuticals, Inc. Results posted | Completed | Phase 3 | June 14, 2018 |
| Pegcetacoplan (APL-2) in Neovascular AMD NCT03465709 Apellis Pharmaceuticals, Inc. Results posted | Terminated | Phase 1, Phase 2 | February 14, 2018 |
| Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects. NCT02588833 Apellis Pharmaceuticals, Inc. Results posted | Completed | Phase 1 | December 1, 2015 |
| Study of Pegcetacoplan (APL-2) Therapy in Patients With Geographic Atrophy NCT02503332 Apellis Pharmaceuticals, Inc. Results posted | Completed | Phase 2 | September 24, 2015 |
| A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH NCT02264639 Apellis Pharmaceuticals, Inc. Results posted | Completed | Phase 1 | February 23, 2015 |
| Assessment of Safety, Tolerability and Pharmacokinetics of Intravitreal Pegcetacoplan (APL-2) for Patients With Wet AMD NCT02461771 Apellis Pharmaceuticals, Inc. Results posted | Completed | Phase 1 | January 28, 2015 |
| C3G/Primary IC-MPGN EAP NCT04729062 Apellis Pharmaceuticals, Inc. | Approved for marketing | — | — |
All 38 studies on ClinicalTrials.gov ↗
Source: ClinicalTrials.gov · verified October 30, 2026
Adverse event reports
- Reports
- 1,783
- Serious
- 1,182
- With death outcome
- 103
Most reported reactions
- Fatigue 160
- Off label use 144
- Visual impairment 138
- Haemoglobin decreased 125
- Vitreous floaters 112
- Haemolysis 107
- Breakthrough haemolysis 91
- Headache 87
- Uveitis 79
- Vitritis 73
- Drug ineffective 69
- Product dose omission issue 67
Adverse event reports do not prove that the substance caused these effects. Side effects not listed here may still occur. Reaction terms are MedDRA terms as reported to FAERS.
Source: FDA FAERS (FAERS data as of July 30, 2026) · verified October 30, 2026 · API query
FDA prescribing information
Official US prescribing information. Dosing sections are intentionally not shown.
Boxed warning Empaveli · August 21, 2026
Original FDA text (English) WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA EMPAVELI, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae , Neisseria meningitidis , and Haemophilus influenzae type B [see Warnings and Precautions (5.1) ] . Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update vaccination for encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, unless the risks of delaying therapy with EMPAVELI outweigh the risk of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria in patients receiving a complement inhibitor. See Warnings and Precautions (5.1) for additional guidance on the management of the risk of serious infections caused by encapsulated bacteria. Patients receiving EMPAVELI are at increased risk for invasive disease caused by encapsulated bacteria, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected. Because of the risk of serious infections caused by encapsulated bacteria, EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the EMPAVELI REMS [see Warnings and Precautions (5.2) ] . WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA See full prescribing information for complete boxed warning. EMPAVELI increases the risk of serious and life-threatening infections caused by encapsulated bacteria including Streptococcus pneumoniae , Neisseria meningitidis and Haemophilus influenzae type B. Complete or update vaccination for encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, unless the risks of delaying EMPAVELI outweigh the risks of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria in patients receiving a complement inhibitor. ( 5.1 ) Patients receiving EMPAVELI are at increased risk for invasive disease caused by encapsulated bacteria, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected. ( 5.1 ) EMPAVELI is available only through a restricted program called EMPAVELI REMS.
- Empaveli HUMAN PRESCRIPTION DRUG Apellis Pharmaceuticals, Inc. August 21, 2026
- Syfovre HUMAN PRESCRIPTION DRUG Apellis Pharmaceuticals, Inc. September 16, 2025
Source: DailyMed / openFDA
Change history
No changes since monitoring began on October 30, 2026.