RegulationPriority Review (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Australia), Priority Review (Australia), Orphan Drug (United Kingdom)

Structure/Sequence

Sequence Code 550935032

Source: *****

Clinical Trials associated with Pegcetacoplan

NCT06961370

/ RecruitingPhase 1

A Phase I, Multi-center, Multi-part Study to Investigate Safety, Tolerability, PK, PD, and Immunogenicity of RO7669330 Intravitreal Injections in Participants With GA Secondary to AMD: Part 1A: Open-label, MAD; Part 1B: Randomized PD Pilot; Part 2: Masked, Randomized, Active-comparator-controlled

The main purpose of this study is to assess the ocular and systemic safety and tolerability of RO7669330 in GA secondary to AMD after multiple unilateral intravitreal (IVT) doses.

Start Date21 May 2025

Sponsor / Collaborator

Roche Holding AG

NCT06161584

/ RecruitingNot Applicable

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

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

Start Date28 Sep 2023

Sponsor / Collaborator

Apellis Pharmaceuticals, Inc.

JPRN-jRCT2041230022

/ RecruitingPhase 3IIT

A PHASE 3, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLINDED, MULTICENTER STUDY TO EVALUATE THE EFFICACY AND SAFETY OF PEGCETACOPLAN IN PATIENTS WITH C3 GLOMERULOPATHY OR IMMUNE-COMPLEX MEMBRANOPROLIFERATIVE GLOMERULONEPHRITIS - A PHASE 3 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF PEGCETACOPLAN IN PATIENTS WITH C3 GLOMERULOPATHY OR IMMUNE-COMPLEX MEMBRANOPROLIFERATIVE GLOMERULONEPHRITIS

Start Date18 Aug 2023

Sponsor / Collaborator-

100 Clinical Results associated with Pegcetacoplan

100 Translational Medicine associated with Pegcetacoplan

100 Patents (Medical) associated with Pegcetacoplan

193

Literatures (Medical) associated with Pegcetacoplan

01 Aug 2025·AMERICAN JOURNAL OF OPHTHALMOLOGY

Ocular Adverse Events Associated with Pegcetacoplan and Avacincaptad Pegol for Geographic Atrophy: A Population-Based Pharmacovigilance Study

Article

Author: Kertes, Peter J ; Muni, Rajeev H ; Popovic, Marko M ; Mihalache, Andrew ; Kailani, Zeena

OBJECTIVE:

To evaluate the postmarketing ocular adverse events (AEs) reported for avacincaptad pegol and pegcetacoplan, the only Food and Drug Administration (FDA)-approved treatments for geographic atrophy (GA).

DESIGN:

Retrospective pharmacovigilance analysis.

SUBJECTS:

Ocular AE reports in the FDA Adverse Event Reporting System (FAERS) in which pegcetacoplan or avacincaptad pegol was identified as the primary suspect drug were analyzed.

METHODS:

Using the OpenVigil 2.1 data mining software, we conducted a retrospective pharmacovigilance analysis of the FAERS database from inception to December 2024. We conducted disproportionality analyses to assess reporting odds ratios (RORs) for specific drug-AE combinations compared with all other drugs in the database.

MAIN OUTCOME MEASURES:

Ocular AEs were evaluated.

RESULTS:

A total of 752 and 80 patients with AEs secondary to pegcetacoplan and avacincaptad pegol, respectively, were identified. Ocular AEs disproportionately overreported for pegcetacoplan included anterior segment (iris) hemorrhage (ROR 1767, 95% CI 538-5803), iris neovascularization (ROR 1248, 95% CI 502-3099), choroidal neovascularization (ROR 1328, 95% CI 956-1845), intraocular injection complication (ROR 2552, 95% CI 1607-4053), hemorrhagic occlusive retinal vasculitis (ROR 4606, 95% CI 2000-10,611), retinal occlusive vasculitis (ROR 2352, 95% CI 1313-4212), and bacterial endophthalmitis (ROR 1260, 95% CI 613-2589). Ocular AEs disproportionately overreported for avacincaptad pegol included choroidal neovascularization (ROR 1169, 95% CI 426-3205), vitritis (ROR 782, 95% CI 316-1936), dry age-related macular degeneration (ROR 684, 95% CI 316-1936), and cystoid macular edema (ROR 445, 95% CI 140-1412).

CONCLUSIONS:

Current prescribing patterns indicate that a broader spectrum of ocular AEs were reported for pegcetacoplan than avacincaptad pegol. These findings aim to enhance clinicians' understanding of the safety profiles of these agents, enabling informed patient care and heightened vigilance of these novel GA treatments.

01 Aug 2025·AMERICAN JOURNAL OF OPHTHALMOLOGY

Pegcetacoplan Treatment for Geographic Atrophy in Age-Related Macular Degeneration Over 36 Months: Data From OAKS, DERBY, and GALE

Article

Author: Boyer, David ; Abbey, Ashkan M ; Heier, Jeffrey ; Holz, Frank G ; Loewenstein, Anat ; Lin, Jason ; Mones, Jordi ; Li, Chao ; Wykoff, Charles C ; Dhoot, Dilsher S ; Vilupuru, Abhi ; Vajzovic, Lejla ; Baumal, Caroline R ; Singerman, Lawrence J ; Chiang, Allen

PURPOSE:

To report 12-month results from the GALE open-label extension study (NCT04770545), evaluating up to 36 months of intravitreal pegcetacoplan treatment for geographic atrophy (GA) in age-related macular degeneration (AMD).

DESIGN:

GALE is a prospective open-label extension study following the 24-month, sham-controlled, phase 3 OAKS (NCT03525613) and DERBY (NCT03525600) studies of pegcetacoplan.

PARTICIPANTS:

Patients with nonsubfoveal or subfoveal GA who completed OAKS, DERBY, or phase 1b APL2-103 (NCT03777332) studies.

METHODS:

Pegcetacoplan was administered monthly (PM) or every other month (PEOM) to all study eyes in GALE. Eyes receiving pegcetacoplan in OAKS and DERBY continued the same regimen (PM-PM and PEOM-PEOM), while eyes observed with sham in OAKS and DERBY crossed over to receive pegcetacoplan at the same dosing interval in GALE (SM-PM and SEOM-PEOM). Safety and efficacy through the first 12 months of GALE were assessed, reflecting up to 36 months of continuous pegcetacoplan treatment.

MAIN OUTCOME MEASURE:

Mean rate of change in GA area, total number of microperimetry scotomatous points, and adverse events.

RESULTS:

Through the first 12 months of GALE, 92.0% (727/790) patient retention was observed. Across all eyes, including eyes with nonsubfoveal and subfoveal GA, pegcetacoplan reduced the mean rate of change in GA area up to 32% versus projected sham. Year after year, the reductions in the mean rate of change in GA area increased, with up to a 42% reduction observed in eyes with nonsubfoveal GA in the PM-PM group compared with projected sham in the first year of GALE. An 18% reduction in new scotomatous points (P = .0156) was observed with PM-PM at 36 months, highlighting a significant impact in a prespecified microperimetry analysis. Adverse events included 33 (4.5%) eyes with exudative AMD, 15 (1.9%) intraocular inflammation (classified as mild or moderate in severity), 1 (0.1%) ischemic optic neuropathy, and 1 (0.1%) infectious endophthalmitis. No events of vasculitis were reported.

CONCLUSION:

Over 36 months, pegcetacoplan continued to reduce GA growth with increasing efficacy over time and reduced formation of new scotomatous points. The safety profile of pegcetacoplan in the first 12 months of GALE was consistent with the prior 24-month OAKS and DERBY studies.

01 Jul 2025·BLOOD CELLS MOLECULES AND DISEASES

Pegcetacoplan for the treatment of warm autoimmune hemolytic anemia: a case report

Letter

Author: Venou, Theodora Maria ; Vlantos, Vasileios Theodoros ; Gavriilaki, Eleni ; Mainou, Maria ; Evangelidis, Paschalis ; Vlachaki, Efthymia

316

News (Medical) associated with Pegcetacoplan

12 Jun 2025

·www.prnewswire.com

Sobi share new clinical data across multiple hematologic diseases at EHA 2025

STOCKHOLM, June 12, 2025 /PRNewswire/ -- Sobi® (STO: SOBI) will present data at the 30th EHA (European Haematology Association) hybrid congress, in Milan, Italy (12-15 June). The congress will feature the latest advances in the treatment of diffuse large B-cell lymphoma (DLBCL), immune thrombocytopenia (ITP), myelofibrosis, paroxysmal nocturnal haemoglobinuria (PNH), and primary hemophagocytic lymphohistiocytosis (pHLH). An extensive programme of poster presentations will showcase Sobi's commitment to helping patients with rare diseases by advancing treatment options. In addition, Sobi will host several scientific symposiums at the congress including: Advancing Therapeutic Knowledge in Paroxysmal Nocturnal Haemoglobinuria: Reshaping disease management to unlock new norms, Thursday, 12 June, 10:00am - 11:30am CEST, at Amber Hall 3 & 4. Boosting Platelets: Expert Approaches to Adult Immune Thrombocytopenia (ITP), Saturday 14 June, 8.00 am - 9.30 am CEST, at Amber Hall 7 & 8. Dissecting Treatment Sequencing in relapsed/refractory DLBCL, from laboratory to real life. Saturday, 14 June, 8:00 am – 9:30 am, CEST at Coral Hall 1. "The breadth of data that we share at this year's EHA congress demonstrates Sobi's comprehensive approach to addressing rare conditions in haematology. We are proud to contribute to advancing the science in several indications from early clinical phases in diffuse large B-cell lymphoma to clinical and real-world evidence in myelofibrosis, primary hemophagocytic lymphohistiocytosis, immune thrombocytopenia and paroxysmal nocturnal haemoglobinuria," said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. Key data to be presented at EHA 2025 About pegcetacoplan in rare diseases Pegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is under investigation for rare diseases across haematology and nephrology. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) as EMPAVELI®/Aspaveli® in the United States, European Union, and other countries globally. About Doptelet® (avatrombopag) Doptelet® (avatrombopag) is indicated for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments, and a treatment of severe thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo an invasive procedure. About Zynlonta® (loncastuximab tesirine) Zynlonta® (loncastuximab tesirine) is a CD19-directed antibody drug conjugate (ADC). Zynlonta as monotherapy is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL), after two or more lines of systemic therapy. About Sobi® Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi's share (STO: SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision . The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

ImmunotherapyDrug Approval

12 Jun 2025

·www.sobi.com

Sobi share new clinical data across multiple hematologic diseases at EHA 2025

Sobi® (STO: SOBI) will present data at the 30th EHA (European Haematology Association) hybrid congress, in Milan, Italy (12-15 June). The congress will feature the latest advances in the treatment of diffuse large B-cell lymphoma (DLBCL), immune thrombocytopenia (ITP), myelofibrosis, paroxysmal nocturnal haemoglobinuria (PNH), and primary hemophagocytic lymphohistiocytosis (pHLH). An extensive programme of poster presentations will showcase Sobi’s commitment to helping patients with rare diseases by advancing treatment options. In addition, Sobi will host several scientific symposiums at the congress including: 1.Advancing Therapeutic Knowledge in Paroxysmal Nocturnal Haemoglobinuria: Reshaping disease management to unlock new norms, Thursday, 12 June, 10:00am - 11:30am CEST, at Amber Hall 3 & 4. 2. Boosting Platelets: Expert Approaches to Adult Immune Thrombocytopenia (ITP), Saturday 14 June, 8.00 am - 9.30 am CEST, at Amber Hall 7 & 8. 3. Dissecting Treatment Sequencing in relapsed/refractory DLBCL, from laboratory to real life. Saturday, 14 June, 8:00 am – 9:30 am, CEST at Coral Hall 1. “The breadth of data that we share at this year’s EHA congress demonstrates Sobi’s comprehensive approach to addressing rare conditions in haematology. We are proud to contribute to advancing the science in several indications from early clinical phases in diffuse large B-cell lymphoma to clinical and real-world evidence in myelofibrosis, primary hemophagocytic lymphohistiocytosis, immune thrombocytopenia and paroxysmal nocturnal haemoglobinuria,” said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. Key data to be presented at EHA 2025 DLBCL PS1911: Initial Results From LOTIS-7: A Phase 1b Study of Loncastuximab Tesirine Plus Glofitamab in Patients With Relapsed/Refractory (R/R) Diffuse Large BCell Lymphoma (DLBCL) Presenting Author: Juan Pablo Alderuccio Poster presentation Session title: Poster Session 2Session date: Saturday, 14 June Session time: 18:30-19:30 CEST Location: Poster Hall PS1957: Updated Safety Run-in Results from LOTIS-5: A Phase 3, Randomized Trial of Loncastuximab Tesirine with Rituximab Versus Immunochemotherapy in Patients With R/R DLBCL Presenting Author: Carmelo Carlo-Stella Immune Thrombocytopenia (ITP) PF1236: Platelet Response to Avatrombopag Among Patients with Primary Immune Thrombocytopenia Who Switched from Eltrombopag or Romiplostim: the REAL-AVA 2.0 Real-World Study Presenting Author: Shruti Chaturvedi Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PF1239: Durability of Response to Avatrombopag Among Patients with Primary Immune Thrombocytopenia: The REAL-AVA 2.0 Real-World Study Presenting Author: Srikanth Nagalla PF1251: Clinically Meaningful Response to Avatrombopag: A Phase 3B Trial for Treatment of Children with ITP Presenting Author: Rachael F. Grace PS2231: Effectiveness and safety of avatrombopag for the treatment of adults with newly diagnosed, persistent, or chronic immune thrombocytopenia: Interim results from the phase 4 ADOPT study Presenting Author: Waleed Ghanima Poster presentation Session title: Poster Session 2Session date: Saturday, 14 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PS2234: Efficacy and safety of avatrombopag for the treatment of pediatric immune thrombocytopenia in the open-label extension of a phase 3, randomized, double-blind, placebo-controlled trial Presenting Author: Rachael F. Grace PS2239: Real-World Treatment Patterns & Clinical Outcomes in Thrombopoietin Receptor Agonist Naive Patients with Immune Thrombocytopenia Treated with Avatrombopag: Interim Results from the REAL-AVA 3.0 Study Presenting Author: Sandhya Panch PS2242: Effectiveness and safety of avatrombopag for treatment of immune thrombocytopenia in older patients and those with comorbidities or prior TPO-RA exposure: Interim results from the phase 4 ADOPT study Presenting Author: María Eva Mingot-Castellano PS2244: Response to Avatrombopag Among Patients with Chronic and Persistent Primary Immune Thrombocytopenia: the REAL-AVA 2.0 Real-World Study Presenting Author: M Y Levy PS2250: Evaluation of Efficacy and Safety of Avatrombopag in Children with Immune Thrombocytopenia based on Disease Duration: Results from the Avatrombopag Phase 3-b Pediatric Trial Presenting Author: Rachael F. Grace PB3676: Baseline Correlates with Durability of Avatrombopag Response: A Phase 3B Trial for Treatment of Children with ITP Publication Only Published on May 14 at 15:30 CEST PB3684: Consistent Response to Avatrombopag across Various Baseline Characteristics: Results from the Phase 3-b Trial for the Treatment of Children with Immune Thrombocytopenia Myelofibrosis PF849: Hematologic improvement experienced by pacritinib-treated patients with myelofibrosis in real-world clinical settings Presenting Author: Michael Marrone Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PF1242: Efficacy of pacritinib vs momelotinib in patients with thrombocytopenic MF: a matched adjusted indicated treatment comparison Presenting Author: Koo Wilson PF1306: Transfusion-related cost and time burden offsets in patients with myelofibrosis treated with pacritinib compared to best available therapy based on PERSIST-2 trial Presenting Author: Abiola Oladapo PS1827: Real-world effectiveness of pacritinib in patients with myelofibrosis who have concurrent thrombocytopenia and anemia Presenting Author: Raajit Rampal Poster presentation Session title: Poster Session 2 Session date: Saturday, 14 June Session time: 18:30-19:30 CEST Location: Poster Hall PS1842: Real-World Treatment Patterns and Clinical Outcomes in Patients with Myelofibrosis Treated with Pacritinib (PAC) with platelets ≥50 x109/L at PAC initiation: Interim results from the MY-PAC Study Presenting Author: Doug Tremblay PS2295: Economic Burden of Cytopenia in Patients with Myelofibrosis: Analysis of a US National Administrative Claims Database Presenting Author: Lucia Marasova PB3079: Cytopenia is associated with real-world disease progression and diminished survival in patients with myelofibrosis: Analysis of a US national administrative claims database Publication Only Published on May 14 at 15:30 CEST Paroxysmal Nocturnal Hemoglobinuria PF672: Early response in complement inhibitor naïve patients with paroxysmal nocturnal hemoglobinuria treated with pegcetacoplan in the Phase 3 PRINCE trial Presenting Author: Austin Kulasekararaj Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PF676: Interim analysis of the ongoing COMPLETE study on the real-world effectiveness of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria (PNH) Presenting Author: Regis Peffault de Latour PS1662: A benefit assessment of pegcetacoplan dose increase in the Phase 3 PEGASUS trial of PNH patients with difficult-to-control disease Presenting Author: Morag Griffin Poster presentation Session title: Poster Session 2Session date: Saturday, 14 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PS1665: Benefit of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria irrespective of baseline transfusion status Presenting Author: Britta Höchsmann Primary Hemophagocytic Lymphohistiocytosis (pHLH) PF1036: Emapalumab in patients with primary hemophagocytic lymphohistiocytosis: Efficacy and safety outcomes from a multinational, open-label, single-arm study Presenting Author: Franco Locatelli Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall About pegcetacoplan in rare diseases Pegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is under investigation for rare diseases across haematology and nephrology. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) as EMPAVELI®/Aspaveli® in the United States, European Union, and other countries globally About Doptelet® (avatrombopag) Doptelet® (avatrombopag) is indicated for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments, and a treatment of severe thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo an invasive procedure. About Zynlonta® (loncastuximab tesirine)Zynlonta® (loncastuximab tesirine) is a CD19-directed antibody drug conjugate (ADC). Zynlonta as monotherapy is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL), after two or more lines of systemic therapy. About Sobi® Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. We provide access to innovative treatments that transform life for people. Our therapies are concentrated within the areas of Haematology, Immunology and Specialty Care. We contribute to societies by improving access to treatment of rare diseases. Every day, we work actively to find better ways to understand and meet patient needs. Find out more about our business and financial performance. Here we present current and past media releases, images and videos.

Phase 3ImmunotherapyClinical ResultDrug ApprovalPhase 1

10 Jun 2025

·pmlive.com

Sobi/Apellis’ pegcetacoplan shows sustained efficacy in phase 3 kidney disease study

Sobi and Apellis Pharmaceuticals have shared positive new data from a late-stage study of their targeted C3 therapy in rare kidney disease patients. The phase 3 VALIANT trial has been evaluating pegcetacoplan in over 120 patients aged 12 years and older with C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). Results from the study’s open-label period, presented at this year’s European Renal Association Congress, showed that pegcetacoplan reduced excess protein in the urine (proteinuria) by 68% compared to placebo at week 26, with this benefit sustained at one year. Pegcetacoplan-treated patients also continued to achieve stabilisation of kidney function, as measured by estimated glomerular filtration rate, and the drug demonstrated similar benefits in patients who switched from placebo to pegcetacoplan at the beginning of the open-label period. C3G and primary IC-MPGN are estimated to affect 5,000 people in the US and up to 8,000 in Europe. Excessive deposits of the C3 protein are a key marker of disease activity in both conditions, and approximately 50% of patients experience kidney failure within five to ten years of diagnosis. Pegcetacoplan is designed to regulate excessive activation of the complement cascade, a part of the body’s immune system, and already holds approvals to treat the rare blood disorder paroxysmal nocturnal haemoglobinuria under the brand names Empaveli and Aspaveli. Presenting author and co-lead principal investigator for VALIANT, Fadi Fakhouri, CHUV Lausanne, said: “Given the high risk of kidney failure, treatment efficacy is incredibly important to C3G and primary IC-MPGN patients, many of whom are in the prime of their lives. This data further underscores the potential of [pegcetacoplan] to make a meaningful difference for patients.” Pegcetacoplan has already been granted Priority Review designation by the US Food and Drug Administration for C3G and primary IC-MPGN, with a target action date of 28 July 2025. The European Medicines Agency has also validated an indication extension application for pegcetacoplan in both diseases. Sobi has exclusive commercialisation rights for systemic pegcetacoplan outside the US, where Apellis has exclusive rights. Apellis also holds global commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy.

Phase 3Clinical ResultPriority ReviewDrug ApprovalNDA

100 Deals associated with Pegcetacoplan

External Link

KEGG	Wiki	ATC	Drug Bank
D11613	Pegcetacoplan	B03XA	-

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Geographic Atrophy	United States	Apellis Pharmaceuticals, Inc.	17 Feb 2023
Hemoglobinuria, Paroxysmal	United States	Apellis Pharmaceuticals, Inc.	14 May 2021

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
C3 glomerulopathy	NDA/BLA	European Union	Apellis Pharmaceuticals, Inc.	20 Feb 2025
C3 glomerulopathy	NDA/BLA	European Union	Swedish Orphan Biovitrum AB	20 Feb 2025
Glomerulonephritis, Membranoproliferative	NDA/BLA	European Union	Apellis Pharmaceuticals, Inc.	20 Feb 2025
Glomerulonephritis, Membranoproliferative	NDA/BLA	European Union	Swedish Orphan Biovitrum AB	20 Feb 2025
Delayed Graft Function	Phase 3	-	Apellis Pharmaceuticals, Inc.	01 Sep 2025
Kidney Failure, Chronic	Phase 3	-	Apellis Pharmaceuticals, Inc.	01 Sep 2025
Cold Agglutinin Disease	Phase 3	United States	Swedish Orphan Biovitrum AB	20 Oct 2022
Cold Agglutinin Disease	Phase 3	Japan	Swedish Orphan Biovitrum AB	20 Oct 2022
Cold Agglutinin Disease	Phase 3	Austria	Swedish Orphan Biovitrum AB	20 Oct 2022
Cold Agglutinin Disease	Phase 3	Belgium	Swedish Orphan Biovitrum AB	20 Oct 2022

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04770545 (OAKS, DERBY, and GALE, Pubmed \| Am J Ophthalmol)	Phase 3	Age Related Macular Degeneration	790	Pegcetacoplan monthly	ywqpbyexnu(vnrnnszkpe) = 33 (4.5%) eyes with exudative AMD pgwygfvusx (gkwlqohwjw ) View more	Positive	01 Aug 2025
NCT03500549 (Pubmed \| Eur J Haematol)	Phase 3	Hemoglobinuria, Paroxysmal complement 5 inhibitors (C5i; eculizumab, ravulizumab)	-	Pegcetacoplan	xscqqwnlab(bsggumjjmm) = nnpbxufuax umcxcffzvd (ubaosgjydj, -90.02 to 21.21)	Positive	25 Apr 2025
NCT04579666 (CTgov)	Phase 2	Amyotrophic Lateral Sclerosis	249	RTP+Pegcetacoplan (RTP: Pegcetacoplan)	ditveiuxna(stzmnnlarr) = ripbbiltun qjplqygtxd (cdznewsfby, 4.71) View more	-	24 Apr 2025
				placebo+pegcetacoplan (RTP: Placebo)	ditveiuxna(stzmnnlarr) = hhkppmamum qjplqygtxd (cdznewsfby, 6.89) View more
NCT03453619 (CTgov)	Phase 2	Idiopathic Membranous Glomerulonephritis \| Glomerulonephritis, Membranoproliferative \| C3 glomerulopathy ... View more	21	pegcetacoplan (Part A: IgAN Subjects)	rovhydrjrg(hiofcnxfhc) = fbwabsekep dtxuwctvjz (hpifrsrium, ighfecjaoh - rbrrnwmkpa) View more	-	13 Feb 2025
				pegcetacoplan (Part A: LN Subjects)	rovhydrjrg(hiofcnxfhc) = pwxxctqkdk dtxuwctvjz (hpifrsrium, kwmxuaoigx - msvpcarzhn) View more
NCT04572854 (NOBLE, CTgov)	Phase 2	Glomerulonephritis, Membranoproliferative \| C3 glomerulopathy \| glomerulonephritis chronic complement component 3 glomerulopathy	13	pegcetacoplan (Part A: Controlled Portion: Group 1)	thlrwmurua = wlkbphqayw zyewgebery (ctxcqqeykq, tcklyufllz - cbwkdgnali) View more	-	07 Feb 2025
				pegcetacoplan (Part A: Controlled Portion: Group 2)	thlrwmurua = rrmwbbuvyv zyewgebery (ctxcqqeykq, pebvpjqjso - hqyfhzshlj) View more
NCT03226678 (CTgov)	Phase 2	Cold Agglutinin Disease \| Warm autoimmune hemolytic anemia	24	wAIHA (Part A: Cohort 1 wAIHA - 270 mg)	ndoqgyiatq = rltqssynpt xadrtgcors (slfdtrbquv, ahabtdrxbi - vgrssisrrw) View more	-	12 Dec 2024
				wAIHA (Part A: Cohort 1 wAIHA - 360 mg)	ndoqgyiatq = fibikhvipo xadrtgcors (slfdtrbquv, vmnqcuromk - doliyiudhy) View more
ASH2024	Not Applicable	Hemoglobinuria, Paroxysmal	-	Complement Inhibitor Experienced	chxudwmdmq(dtogyxbbrz) = ctykssjzso ccvqvdgtfh (dipsnlkpcg, 1.7) View more	-	09 Dec 2024
				Complement Inhibitor Naïve	chxudwmdmq(dtogyxbbrz) = vazobjeldi ccvqvdgtfh (dipsnlkpcg, 1.3) View more
ASH2024	Not Applicable	Hemoglobinuria, Paroxysmal	-	Pegcetacoplan	cdojdsxzwd(fxllpgdryt) = 13 patients experienced 1 to 3 acute hemolytic events (n=20, median 1 per patient, IQR [1; 2]) with hospitalization required to manage 6 events odclokmndr (pkciclqrqv ) View more	-	07 Dec 2024
EURETINA2024	Not Applicable	Geographic Atrophy	-	Pegcetacoplan 15 mg per 0.1 mL intravitreal injection monthly	zmprwhdsjt(cyuzpxxpfd) = mqjnbgovni fkawcrnqms (fxczdvasvg, 0.09)	-	19 Sep 2024
				Pegcetacoplan 15 mg per 0.1 mL intravitreal injection every other month	zmprwhdsjt(cyuzpxxpfd) = aurwetqzcw fkawcrnqms (fxczdvasvg, 0.09)
EURETINA2024	Not Applicable	Geographic Atrophy	-	Pegcetacoplan monthly (PM)	sdujnheuyn(xkwfyfusho) = the estimated rate of reported events of retinal vasculitis in the post-marketing setting remaining <0.01% per injection with an estimated 215,000 injections administered in the real-world setting through February 2024 xfdhxrvnez (yksbseqxfp )	-	19 Sep 2024
				Pegcetacoplan every other month (PEOM)

Translational Medicine

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Deal

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Core Patent

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Clinical Trial

Identify the latest clinical trials across global registries.

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Approval

Accelerate your research with the latest regulatory approval information.

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Biosimilar

Competitive landscape of biosimilars in different countries/locations. Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3.

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Regulation

Understand key drug designations in just a few clicks with Synapse.

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Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis