A Phase 3, Randomized, Placebo-Controlled, Double-Blinded, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults at High Risk of Delayed Graft Function (DGF) Following Kidney Allograft Transplantation

A PHASE 3 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF PEGCETACOPLAN IN ADULTS AT HIGH RISK OF DELAYED GRAFT FUNCTION (DGF) FOLLOWING KIDNEY ALLOGRAFT TRANSPLANTATION

开始日期2025-09-01

申办/合作机构

Apellis Pharmaceuticals, Inc.

NCT06961370

/ Recruiting临床1期

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.

开始日期2025-07-16

申办/合作机构

Roche Holding AG

NCT06161584

/ RecruitingN/A

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

开始日期2023-09-28

申办/合作机构

Apellis Pharmaceuticals, Inc.

100 项与 Pegcetacoplan 相关的临床结果

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100 项与 Pegcetacoplan 相关的转化医学

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100 项与 Pegcetacoplan 相关的专利（医药）

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201

项与 Pegcetacoplan 相关的文献（医药）

2025-09-01·AMERICAN JOURNAL OF OPHTHALMOLOGY

Visual Acuity and Quality of Life Outcomes With Pegcetacoplan Treatment: A Post Hoc Analysis From the OAKS and DERBY Trials

Article

作者: Jones, Daniel L ; Baumal, Caroline R ; Intorcia, Michele ; McKeown, Alex ; Sarda, Sujata P ; Sheidow, Tom ; Garg, Sunir ; Stevens, Warren ; Davis, Matthew ; Chiang, Allen ; Schmidt-Erfurth, Ursula M

OBJECTIVE:

To evaluate the effect of pegcetacoplan on visual function and patient quality of life (QoL) measures based on distance of geographic atrophy (GA) lesions from the center of the fovea.

DESIGN:

Post hoc analysis from OAKS and DERBY, two global, 24-month, multicenter, randomized, double-masked, sham-controlled phase 3 studies evaluating pegcetacoplan treatment for GA in age-related macular degeneration (AMD).

PARTICIPANTS:

888 study patients with GA for whom all data necessary for the post hoc analysis to be performed had been collected were included.

METHODS:

Best-corrected visual acuity (BCVA) and 25-item National Eye Institute Visual Functioning Questionnaire (NEI VFQ-25) were assessed at baseline and every 4 and 6 months, respectively, to completion at month 24 in both pegcetacoplan-treated eyes and sham (observed) eyes. Eyes were stratified based on optical coherence tomography‒derived GA lesion location ≥250 µm (n = 192) or <250 µm (n = 696) from the foveal center.

MAIN OUTCOME MEASURES:

Change from baseline in BCVA and NEI VFQ-25 was evaluated over 24 months in GA lesions based on distance (≥250 or <250 µm) from the foveal center. Adjustment was conducted via propensity score weighting, where model specification and baseline covariate selection were done a priori based on clinical rationale.

RESULTS:

Pegcetacoplan-treated eyes with GA lesion margins ≥250 µm from the foveal center demonstrated directionally slower decline in visual acuity (mean +5.6 [SE 3.2] [P = .0785]) and QoL (mean +4.0 [SE 2.4] [P = .0905]) from baseline at 24 months compared with sham. The change in visual acuity (BCVA, mean -1.6 [SE 1.1] [P = .1522]) and QoL (NEI VFQ-25, -2.3 [1.1] [P = .0284]) in pegcetacoplan-treated eyes with GA lesion margins <250 µm from the foveal center were within the limits of variability compared with sham.

CONCLUSIONS:

Pegcetacoplan treatment demonstrated a potentially meaningful slower decline in visual acuity and QoL for patients with GA lesions ≥250 µm from the foveal center. The change in visual acuity and QoL seen with pegcetacoplan treatment for patients with GA lesions <250 µm from the foveal center were within the limits of variability.

2025-08-01·EUROPEAN JOURNAL OF HAEMATOLOGY

Anchored Indirect Treatment Comparison Finds Comparable Effects of Pegcetacoplan and Iptacopan in Paroxysmal Nocturnal Haemoglobinuria

Article

作者: Czech, Barbara ; Nazir, Jameel ; Wilson, Koo ; Wojciechowski, Piotr ; Kulasekararaj, Austin ; Szer, Jeff ; Hakimi, Zalmai ; Hillmen, Peter ; Dingli, David ; de Latour, Regis Peffault

ABSTRACT:

Background:

Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra‐rare, acquired non‐malignant haematological disorder characterised by thrombosis risk, serious complications and debilitating symptoms in untreated patients.

Objective:

This anchored indirect treatment comparison (ITC) evaluated efficacy data between proximal complement 3 inhibitor (C3i) pegcetacoplan and factor B inhibitor, iptacopan, in patients with PNH previously treated with complement 5 inhibitors (C5i; eculizumab, ravulizumab).

Methods:

Respective pivotal studies provided patient‐level trial data for pegcetacoplan (16‐week PEGASUS [NCT03500549]) and published data for iptacopan (24‐week APPLY PNH [NCT04558918]). Differences in study design, duration and statistical methods between PEGASUS and APPLY PNH necessitated the comparative analyses to be conducted on secondary measures based on haemoglobin (Hb) levels, absolute reticulocyte count (ARC), lactate dehydrogenase (LDH) levels, and patient‐reported outcomes on the Functional Assessment of Chronic Illness Therapy–Fatigue (FACIT‐Fatigue) scale scores. The availability of a common reference C5i treatment group in both PEGASUS and APPLY PNH studies allowed anchored ITC (Bucher method). Simulated treatment comparison (STC) assessed the robustness of the main analysis.

Results:

Overall, baseline characteristics of the populations in the PEGASUS and APPLY PNH studies were broadly comparable. Anchored ITC showed comparable outcomes (mean difference, [95% CI]) on change‐from‐baseline to end of study for pegcetacoplan versus C5i, and iptacopan versus C5i, respectively, across endpoints: Hb level (−0.49 g/dL [−1.78, 0.80]); ARC (–34.41 × 109/L [−90.02, 21.21]); LDH level (−115.16 U/L [−244.40, 14.01]); FACIT‐Fatigue score (3.57 [−5.60, 12.73]). Finally, the STC produced results consistent with the main Bucher analyses across all clinical endpoints and patient‐reported fatigue, providing similar point estimates and confidence intervals.

Conclusion:

This anchored ITC, based on data from pivotal trials, did not indicate significant differences in clinical or patient‐reported outcomes between pegcetacoplan and iptacopan in PNH treatment. The findings suggest that PNH treatment decisions should also consider individualised disease‐ and patient‐related factors.Trial Registration:ClinicalTrials.gov identifier: NCT03500549.

2025-08-01·AMERICAN JOURNAL OF OPHTHALMOLOGY

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

Article

作者: 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.

435

项与 Pegcetacoplan 相关的新闻（医药）

2025-07-30

·药事纵横

首款！两类罕见肾病疗法获FDA批准

2025年7月28日，Apellis Pharmaceuticals宣布FDA已批准其 EMPAVELI （pegcetacoplan）作为 12 岁及以上患者 C3 肾小球病（C3G）或原发性免疫复合物膜增生性肾小球肾炎（IC-MPGN）的首款治疗药物，以减少患者蛋白尿。C3G和原发性 IC-MPGN 是罕见的肾脏疾病，尽管C3G与原发性IC-MPGN是两种不同的疾病，但二者的潜在病因和疾病进展却极为相似，C3G 和原发性IC-MPGN 的体征和症状包括：血尿、蛋白尿、腿部水肿、高血压、以及尿量减少，若不进行治疗，这些罕见疾病往往会在确诊后5至10年内导致肾功能衰竭，患者需接受透析或肾移植。此外在既往接受过肾移植的患者中，90%会出现疾病复发。EMPAVELI 的获批基于 VALIANT 研究为期六个月的积极结果，该结果在所有三个关键疾病标志物方面均显示出获益：（1）蛋白尿减少：该研究达到了主要终点，与安慰剂相比，接受 EMPAVELI 治疗的患者蛋白尿显著减少 68%（p<0.0001）。（2）肾功能稳定：通过估算肾小球滤过率（eGFR）衡量，接受 EMPAVELI 治疗的患者相较于安慰剂组实现了肾功能稳定（名义 p=0.03）（3）C3 染色减少：与安慰剂相比，大多数接受 EMPAVELI 治疗的患者 C3 染色强度降低（p<0.0001），71%接受 EMPAVELI 治疗的患者 C3 染色强度达到零，表明 C3 沉积完全清除。EMPAVELI 的安全性已得到充分证明，研究中最常见的不良反应（≥10%）是输液部位反应、发热、鼻咽炎、流感、咳嗽和恶心。EMPAVELI是一种聚乙二醇化双环肽，与补体蛋白 C3 及其活化片段 C3b 结合，从而调节 C3 的裂解和补体活化下游效应物的产生，补体级联反应是人体免疫系统的一部分，补体级联反应可导致许多严重疾病的发生和进展。此前EMPAVELI已获批用于阵发性睡眠性血红蛋白尿（PNH）和地图样萎缩（GA），此次获批不仅是C3G和IC-MPGN治疗领域的 “零的突破”，更标志着补体抑制剂从单一疾病治疗向多系统疾病管理的跨越，首次为罕见肾病提供疾病修饰疗法、首次覆盖青少年患者、验证补体系统的跨疾病治疗潜力，这一进展将推动肾病治疗进入精准靶向时代，为更多患者带来希望。信息来源：apellis官网药事纵横投稿须知：稿费已上调，欢迎投稿

临床结果

2025-07-29

·药明康德

首款！潜在重磅疗法再获FDA批准；礼来抗癌小分子达3期临床主要终点……

首款！潜在重磅疗法再获FDA批准Apellis Pharmaceuticals日前宣布，美国FDA已批准Empaveli（pegcetacoplan）用于治疗12岁及以上患有C3肾小球病（C3G）或原发性免疫复合物膜增生性肾小球肾炎（IC-MPGN）的患者，以减少患者的蛋白尿。根据新闻稿，pegcetacoplan是获FDA批准用以治疗这两类罕见肾病的首款疗法。此次批准主要基于3期VALIANT研究的积极数据。研究结果显示，pegcetacoplan治疗组的蛋白尿水平显著降低68%（p<0.0001），达到主要终点，并同时在肾功能稳定性方面也优于安慰剂组（p=0.03）。此外，多数接受pegcetacoplan治疗的患者补体C3染色强度明显下降，其中71%实现了C3染色完全清除（p<0.0001）。这些获益在青少年和成年患者中均表现一致，并涵盖接受器官移植后复发的C3G患者群体，进一步验证了pegcetacoplan在广泛人群中的治疗潜力。Pegcetacoplan是一款靶向C3补体蛋白的聚乙二醇化（PEGylated）双环肽疗法。双环肽分子集抗体、小分子药物及肽类的特性于一身，具有与抗体类似的亲和性和精确的靶向特异性；同时，由于它们分子量较小，使得其能够快速深入地渗透组织。2021年5月，pegcetacoplan获FDA批准用于治疗阵发性睡眠性血红蛋白尿症（PNH）。2023年2月，它获得FDA批准用以治疗由年龄相关性黄斑变性（AMD）引起的地图样萎缩（GA）。值得一提的是，这款药物被行业媒体Evaluate评为2023年有望获批的十大潜在重磅疗法之一。礼来小分子达3期试验主要终点礼来公司（Eli Lilly and Company）今日宣布，其非共价布鲁顿酪氨酸激酶（BTK）抑制剂Jaypirca（pirtobrutinib）在BRUIN CLL-314临床3期试验中取得积极顶线结果。该研究针对慢性淋巴细胞白血病或小淋巴细胞淋巴瘤（CLL/SLL）患者，评估Jaypirca与获批共价BTK抑制剂相比的疗效，入组患者既往接受过治疗但未使用过BTK抑制剂。研究结果显示，试验达到由独立审查委员会（IRC）评估的主要终点——总缓解率（ORR）的非劣效标准，且Jaypirca在ORR方面展现出有利趋势（名义p<0.05）。在关键次要终点方面，无进展生存期（PFS）虽在本次分析中尚不成熟，但呈现出对Jaypirca有利的趋势，相关优效性分析将在后续进一步开展。总生存期（OS）未观察到不利影响。安全性方面，Jaypirca在本研究中的整体安全性与此前临床试验结果一致，进一步支持其作为CLL/SLL治疗新选择的潜力。Jaypirca是一款非共价、具高度选择性的BTK抑制剂。该疗法在2023年1月获得FDA加速批准，用以治疗复发/难治性套细胞淋巴瘤（MCL）患者。2023年12月，这款疗法再获FDA加速批准，用于治疗慢性淋巴细胞白血病或小淋巴细胞淋巴瘤成人患者。1.32亿美元！核药公司完成B轮融资放射性药物公司ARTBIO今日宣布完成1.32亿美元B轮融资。本轮融资由新晋投资方Sofinnova Investments与B Capital联合领投，F-Prime、Omega Funds和Third Rock Ventures等现有投资者继续参与，卡塔尔投资局（Qatar Investment Authority）与Alexandria Venture Investments也作为新投资方参与其中。此次融资将用于加速公司α粒子放射性配体疗法（ARTs）项目的开发进程。ARTBIO表示，本轮资金将重点推动其主打候选药物AB001在转移性去势抵抗性前列腺癌（mCRPC）患者中的2期临床试验，同时推进多个尚未披露项目的IND申报准备。公司还计划进一步拓展其基于专有反应堆技术构建的供应链体系，加速全球临床试验的推进，并为未来的商业化铺路。参考资料：[1] FDA Approves Apellis’ EMPAVELI® (pegcetacoplan) as the First C3G and Primary IC-MPGN Treatment for Patients 12 and Older. Retrieved July 29, 2025 from https://www.globenewswire.com/news-release/2025/07/28/3122935/0/en/FDA-Approves-Apellis-EMPAVELI-pegcetacoplan-as-the-First-C3G-and-Primary-IC-MPGN-Treatment-for-Patients-12-and-Older.html[2] Lilly's Jaypirca (pirtobrutinib), the first and only approved non-covalent (reversible) BTK inhibitor, met its primary endpoint in a head-to-head Phase 3 trial versus Imbruvica (ibrutinib) in CLL/SLL. Retrieved July 29, 2025 from https://www.prnewswire.com/news-releases/lillys-jaypirca-pirtobrutinib-the-first-and-only-approved-non-covalent-reversible-btk-inhibitor-met-its-primary-endpoint-in-a-head-to-head-phase-3-trial-versus-imbruvica-ibrutinib-in-cllsll-302515393.html[3] ARTBIO Announces $132 Million Series B Financing to Advance Pipeline of Alpha Radioligand Therapies and Expand Manufacturing and Supply Chain Infrastructure. Retrieved July 29, 2025 from https://artbio.com/artbio-raises-132m-series-b-to-advance-alpha-radioligand-therapies-and-expand-infrastructure免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

临床3期临床结果加速审批临床2期上市批准

2025-07-29

·PipelineReview

FDA Approves Apellis’ EMPAVELI® (pegcetacoplan) as the First C3G and Primary IC-MPGN Treatment for Patients 12 and Older

WALTHAM, MA, USA I July 28, 2025 I Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the U.S. Food and Drug Administration (FDA) has approved EMPAVELI ® (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older, to reduce proteinuria. C3G and primary IC-MPGN are rare kidney diseases, affecting 5,000 people in the United States. 1 “I’m excited to now have a highly effective therapy for a broad range of patients living with C3G and primary IC-MPGN,” said Carla Nester, M.D., MSA, FASN, lead principal investigator for the VALIANT study, professor of internal medicine and pediatrics and director of pediatric nephrology, University of Iowa Stead Family Children’s Hospital. “With standard of care, patients living with these rare and severe diseases frequently progress to kidney failure, necessitating lifelong dialysis and/or a kidney transplant. Given the urgent need, particularly in children, the approval of EMPAVELI marks a pivotal moment in the treatment of rare kidney diseases.” In the Phase 3 VALIANT study, EMPAVELI demonstrated an unprecedented 68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits as measured by C3 staining, compared to placebo. The positive results were consistent across adolescent and adult patients with C3G and primary IC-MPGN, and in C3G patients with post-transplant disease recurrence. “EMPAVELI has the potential to be truly transformational for patients with C3G and primary IC-MPGN, who until now have had very few treatment options. In the largest pivotal study of these diseases, EMPAVELI demonstrated its potential to preserve kidney function by controlling all three key markers of disease,” said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer, Apellis. “As Apellis’ third approval in four years, this milestone underscores the unique ability of targeting C3 to improve patients’ lives. We are deeply grateful to everyone who made this approval possible and look forward to building on this momentum as we advance pivotal studies of EMPAVELI in other rare kidney diseases.” “The approval of EMPAVELI is a historic milestone for people living with C3G and primary IC-MPGN, many of whom are adolescents or young adults,” said Josh Tarnoff, chief executive officer, NephCure. “We recognize Apellis’ commitment to these patients and their families, and to the research and innovation that will bring this life-changing treatment into the hands of patients that need it most.” The approval of EMPAVELI is based on positive six-month results from the VALIANT study, demonstrating benefits across all three key markers of disease: The safety profile of EMPAVELI is well-established, with >2,200 patient years across all approved indications. The most common adverse reactions in the VALIANT study (≥10%) were infusion site reactions, pyrexia, nasopharyngitis, influenza, cough, and nausea. Apellis is committed to helping patients with treatment access and support. ApellisAssist ® is a program designed to help EMPAVELI patients along their treatment journey by providing a comprehensive system of support including help navigating insurance coverage, financial assistance for eligible patients, disease education, and ongoing product support. Patients and healthcare providers can call 1-888-273-5547 for more information. Conference Call and Webcast Apellis will host a conference call and webcast to discuss the FDA’s approval of EMPAVELI tomorrow, July 29, 2025 at 8:00 a.m. ET. To access the live call by phone, please pre-register for the call here . A live audio webcast of the event and accompanying slides may also be accessed through the “Events and Presentations” page of the “Investors and Media” section of the company’s website . A replay of the webcast will be available for 30 days following the event. About C3 Glomerulopathy (C3G) and Primary Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN) C3G and primary IC-MPGN are rare and debilitating kidney diseases that can lead to kidney failure. Excessive C3 deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. Approximately 50% of people living with C3G and primary IC-MPGN suffer from kidney failure within five to 10 years of diagnosis, requiring a burdensome kidney transplant or lifelong dialysis therapy. 2-4 Additionally, approximately 90% of patients who previously received a kidney transplant will experience disease recurrence. 5 The diseases are estimated to affect 5,000 people in the United States and up to 8,000 in Europe. 1 About the VALIANT Study The VALIANT Phase 3 study ( NCT05067127 ) was a randomized, placebo-controlled, double-blinded, multi-center study that evaluated EMPAVELI® (pegcetacoplan) efficacy and safety in 124 patients who were 12 years of age and older with C3G or primary IC-MPGN. It is the largest single trial conducted in these populations and the only study to include pediatric and adult patients, with native and post-transplant kidneys. Study participants were randomized to receive EMPAVELI or placebo twice weekly for 26 weeks. Following this 26-week randomized controlled period, patients were able to proceed to a 26-week open-label phase in which all patients received EMPAVELI. The primary endpoint of the study was the log transformed ratio of urine protein-to-creatinine ratio (UPCR) at Week 26 compared to baseline. About EMPAVELI ® /Aspaveli ® (pegcetacoplan) EMPAVELI ® /Aspaveli ® (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. It is approved for the treatment of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in the United States and paroxysmal nocturnal hemoglobinuria (PNH) in the United States, European Union, and other countries globally. The therapy is also under investigation for other rare diseases. Please see full Prescribing Information , including Boxed WARNING regarding serious infections caused by encapsulated bacteria, and Medication Guide . About Apellis Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company leading the way in complement science to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two C3-targeting medicines approved to treat four serious diseases. Breakthroughs for patients include the first-ever therapy for geographic atrophy, a leading cause of blindness, and the first treatment for patients 12 and older with C3G or primary IC-MPGN, two severe, rare kidney diseases. We believe we have only begun to unlock the potential of targeting C3 across many serious diseases. For more information, please visit http://apellis.com or follow us on LinkedIn and X . References 1. Data on file using literature consensus. 2. Smith RJH, et al. Nat Rev Nephrol . 2019;15(3):129-143. 3. Servais A, et al. Kidney Int . 2012;82(4):454-464. 4. Zand L, et al. J Am Soc Nephrol . 2014;25(5):1110-1117. 5. Tarragón, B, et al. C3 Glomerulopathy Recurs Early after Kidney Transplantation in Serial Biopsies Performed within the First 2 Years after Transplantation. Clinical Journal of the American Society of Nephrology. August 2024; 19(8)1005-1015 . doi: 10.2215/CJN.0000000000000474. SOURCE: Apellis Pharmaceuticals

临床结果临床3期上市批准

100 项与 Pegcetacoplan 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11613	Pegcetacoplan	B03XA	-

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
C3肾小球病	美国	Apellis Pharmaceuticals, Inc.	2024-10-07
膜增生性肾小球肾炎	美国	Apellis Pharmaceuticals, Inc.	2024-10-07
地图样萎缩	美国	Apellis Pharmaceuticals, Inc.	2023-02-17
阵发性睡眠性血红蛋白尿症	美国	Apellis Pharmaceuticals, Inc.	2021-05-14

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
移植物功能延迟恢复	临床3期	-	Apellis Pharmaceuticals, Inc.	2025-09-01
终末期肾脏病	临床3期	-	Apellis Pharmaceuticals, Inc.	2025-09-01
冷凝集素病	临床3期	美国	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	日本	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	奥地利	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	比利时	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	加拿大	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	芬兰	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	格鲁吉亚	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	德国	Swedish Orphan Biovitrum AB	2022-10-20

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临床结果

适应症

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04770545 (OAKS, DERBY, and GALE, Pubmed \| Am J Ophthalmol)	临床3期	年龄相关性黄斑变性	790	Pegcetacoplan monthly	醖襯夢膚廠窪齋範糧範(獵艱膚構範選廠繭鏇艱) = 33 (4.5%) eyes with exudative AMD 積艱構遞淵鹽艱襯憲鏇 (膚鹽鏇壓觸鹹顧簾鹽鬱 ) 更多	积极	2025-08-01
PF675 (EHA2025)	N/A	C5 inhibitors	26	Pegcetacoplan	糧獵選艱鬱積蓋觸壓淵(構鹹醖蓋鏇製餘襯膚顧) = 窪膚鹽積願壓艱遞糧醖醖遞積鏇餘壓獵鬱衊觸 (膚鹽衊觸築蓋鏇膚壓遞 ) 更多	-	2025-05-14
PS1665 (EHA2025)	临床3期	阵发性睡眠性血红蛋白尿症	-	PEGcetacoplan (PNH patients with <4 RBCs)	糧積夢範衊製鹽壓鏇襯(獵鏇廠積製淵廠構簾獵) = Initial reductions in mean ARC were sustained 淵糧遞鏇夢膚範憲廠憲 (願蓋膚鏇願獵鹹鏇網築 ) 更多	积极	2025-05-14
				PEGcetacoplan (PNH patients with ≥4 RBCs)
NCT05776472 (COMPLETE, EHA2025)	N/A	阵发性睡眠性血红蛋白尿症	70	Pegcetacoplan	選壓壓淵蓋鹹夢鹽範淵(顧遞築鑰憲膚網窪衊窪) = 獵鑰襯襯鬱鏇遞獵膚蓋餘構願艱範鬱衊憲衊艱 (積餘衊選觸選鹽簾廠鏇, 0.8 ~ 3.3) 更多	-	2025-05-14
NCT04085601 (PRINCE, EHA2025)	临床3期	阵发性睡眠性血红蛋白尿症一线	46	PEGCetacoplan	窪糧蓋範遞襯獵淵觸糧(艱衊襯選選願齋夢鹽鏇) = 鏇顧鏇鑰醖製觸膚製夢鏇衊遞製襯膚願積顧製 (壓廠廠醖鹹齋夢艱製製 )	积极	2025-05-14
NCT03500549 (Pubmed \| Eur J Haematol)	临床3期	阵发性睡眠性血红蛋白尿症 complement 5 inhibitors (C5i; eculizumab, ravulizumab)	-	Pegcetacoplan	範鑰鑰製選憲壓蓋糧憲(膚構廠選簾簾襯餘淵廠) = 膚獵齋糧夢構製獵鹽鏇網壓廠膚鑰鏇夢積鏇製 (構觸壓積膚顧築衊構繭, -90.02 ~ 21.21)	积极	2025-04-25
NCT04579666 (CTgov)	临床2期	肌萎缩侧索硬化	249	Pegcetacoplan (RTP: Pegcetacoplan)	衊範憲繭壓顧艱範築築(顧網積製鹽製糧構衊齋) = 廠膚艱簾廠選廠齋襯觸築窪獵壓醖築艱簾餘襯 (積鬱願淵觸顧膚蓋艱繭, 4.71) 更多	-	2025-04-24
				placebo+pegcetacoplan (RTP: Placebo)	衊範憲繭壓顧艱範築築(顧網積製鹽製糧構衊齋) = 齋繭衊糧醖選糧窪製廠築窪獵壓醖築艱簾餘襯 (積鬱願淵觸顧膚蓋艱繭, 6.89) 更多
NCT03453619 (CTgov)	临床2期	特发性膜性肾病 \| 膜增生性肾小球肾炎 \| C3肾小球病 ... 更多	21	pegcetacoplan (Part A: IgAN Subjects)	顧膚觸醖網鹹鬱壓壓製(鑰襯觸壓糧鏇願醖夢蓋) = 積繭夢糧積觸選襯願網觸窪繭鹹鬱築積範觸齋 (繭構築齋憲願窪艱壓構, 膚醖簾廠淵衊襯壓鹽製 ~ 蓋選醖獵鬱獵願廠願構) 更多	-	2025-02-13
				pegcetacoplan (Part A: LN Subjects)	顧膚觸醖網鹹鬱壓壓製(鑰襯觸壓糧鏇願醖夢蓋) = 糧築積膚蓋獵鬱鬱積範觸窪繭鹹鬱築積範觸齋 (繭構築齋憲願窪艱壓構, 鹽糧憲衊繭簾觸簾襯淵 ~ 選艱觸鬱壓憲積製衊窪) 更多
NCT04572854 (NOBLE, CTgov)	临床2期	膜增生性肾小球肾炎 \| C3肾小球病 \| 肾小球肾炎慢性补体成分 3 肾小球病	13	pegcetacoplan (Part A: Controlled Portion: Group 1)	廠鏇憲鑰鬱構範積淵壓 = 夢襯餘鑰鏇憲膚顧淵夢淵夢艱遞鹹簾窪蓋繭鑰 (選鑰餘範夢觸構膚襯糧, 糧蓋憲壓糧遞製築構蓋 ~ 襯選衊繭積獵鬱簾膚簾) 更多	-	2025-02-07
				pegcetacoplan (Part A: Controlled Portion: Group 2)	廠鏇憲鑰鬱構範積淵壓 = 餘鬱醖餘選齋網鬱簾醖淵夢艱遞鹹簾窪蓋繭鑰 (選鑰餘範夢觸構膚襯糧, 艱蓋獵齋艱網膚襯齋顧 ~ 顧鹽淵顧憲製製艱鑰網) 更多
NCT03226678 (CTgov)	临床2期	冷凝集素病 \| 温热型自身免疫性溶血性贫血	24	wAIHA (Part A: Cohort 1 wAIHA - 270 mg)	淵蓋淵範簾顧壓鬱積顧 = 製醖鏇窪觸窪襯網觸鬱鑰鑰範網積淵壓蓋觸繭 (餘網製獵遞繭觸餘簾顧, 鏇鹽蓋鹹繭鹹衊觸壓淵 ~ 範窪蓋製醖廠選鬱憲鑰) 更多	-	2024-12-12
				wAIHA (Part A: Cohort 1 wAIHA - 360 mg)	淵蓋淵範簾顧壓鬱積顧 = 憲鏇獵願鏇遞窪壓積鑰鑰鑰範網積淵壓蓋觸繭 (餘網製獵遞繭觸餘簾顧, 選糧襯衊艱簾繭繭齋簾 ~ 鑰齋醖網壓鏇選簾衊顧) 更多