A 2-year, Single-arm, Multi-center Study to Explore the Efficacy and Safety of Pegcetacoplan in Preventing Disease Recurrence in Adolescent and Adult Kidney Transplant Recipients With C3 Glomerulopathy or Primary Immune-complex Membranoproliferative Glomerulonephritis

To assess the efficacy and safety of pegcetacoplan for the prevention of recurrent C3G or primary IC-MPGN in kidney transplant recipients

开始日期2027-01-21

申办/合作机构

Swedish Orphan Biovitrum AB

NCT07213960

/ Recruiting临床2/3期

A Sequential Phase 2/3, Single-Arm, Open-Label Study in Adults Followed by a Randomized, Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of APL2 in Adults and Adolescents With Focal Segmental Glomerulosclerosis

This is a sequential phase 2/3 study to evaluate the efficacy and safety of twice-weekly subcutaneous (SC) infusions of APL2 in patients diagnosed with FSGS. The initial phase 2 portion is a single-arm, open-label study in adults diagnosed with FSGS. Phase 2 will commence prior to randomizing for phase 3. The phase 3 portion of the study is a randomized, placebo-controlled, double-blinded, multicenter study in adults and adolescents diagnosed with FSGS.

开始日期2026-12-01

申办/合作机构

Apellis Pharmaceuticals, Inc.

NCT07495722

/ Not yet recruiting临床1期IIT

Safely Quenching Complement in Stroke Subjects: A Phase 1 Safety Trial

This study will include adults (ages 18-80) who have had a stroke caused by a large blood clot blocking blood flow in the brain. All patients in the study must have already had a treatment called a thrombectomy, where doctors remove the clot to help blood flow return to the brain.
The goal of this study is to test the safety of a drug called EMPAVELI (pegcetacoplan). This drug is meant to lower swelling and inflammation that can happen after blood flow returns. The hope is that it may help protect the brain from more damage and improve recovery.
People in the study will get three doses of EMPAVELI through an EMPAVELI-designed pump 0-3 hours post thrombectomy surgery and 24 and 48 hours after the initial dose. Doctors will check them with exams, blood tests, brain scans, and other tests while they are there. Patients will also have follow-up visits at 30 and 90 days to see how they are doing, per the usual standard of care.
This research is important because, even with current stroke treatments, many patients still have problems like disability. If this drug is found to be safe, it could lead to better treatments to protect the brain and help people recover more fully after a stroke.

开始日期2026-10-01

申办/合作机构

Columbia University

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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235

项与 Pegcetacoplan 相关的文献（医药）

2026-12-31·Hematology

Real-world clinical outcomes and treatment response in complement inhibitor experienced and naïve PNH patients prescribed pegcetacoplan in Europe and Canada

Article

作者: Panse, Jens ; Kerr, Brianne ; Hatchell, Niall ; Williamson, Sam ; Czech, Barbara ; Wilson, Koo ; Patriquin, Christopher J. ; Taylor, Yasmin ; Llamas, Juan Carlos Vallejo ; Hakimi, Zalmai

OBJECTIVES:

This study investigated outcomes and response to pegcetacoplan in complement inhibitor naïve (CI_naïve) and experienced (CI_exp) PNH patients.

METHODS:

Data were drawn from the Adelphi PNH Disease Specific Programme™, a cross-sectional survey of physicians and their patients in Canada, France, Germany, Italy, Spain, and the UK between December 2023 and April 2024. Physicians reported patient characteristics and clinical outcomes, which were used to define pegcetacoplan response. Results were stratified by prior CI prescription. Analyses were descriptive.

RESULTS:

Forty-eight physicians reported on 63 patients (CI_naïve:11; CI_exp:52). Median (IQR) age was 45.0 (33.0-55.0) years, 60.3% were male. At treatment initiation versus at survey for CI_naïve and CI_exp, respectively, median (IQR) haemoglobin levels (g/dL) were 8.0 (5.0-9.0) versus 12.0 (11.2-12.3), and 9.0 (8.0-9.2) versus 11.0 (10.2-12.0); lactate dehydrogenase levels (U/L) were 500 (350-1625) versus 250 (175-525), and 470 (290-588) versus 265 (190-379); absolute reticulocyte counts (x10⁹ /L) were 220 (151-250) versus 110 (95-140), and 212 (134-281) versus 120 (105-228). At pegcetacoplan initiation, 4.4% of patients met criteria of good-to-complete response versus 79.5% at survey.

CONCLUSIONS:

Pegcetacoplan proved effective for CI_naïve and CI_exp patients in a real-world setting, with numerically improved clinical outcomes and high response rate.

2026-12-31·Hematology

Real-world pegcetacoplan treatment of paroxysmal nocturnal hemoglobinuria patients with an unsatisfactory response to prior therapies

Article

作者: Vara, Míriam ; González, Ataúlfo ; Benet, Carmen ; Díaz, Bianca ; Vallejo, Carlos ; Martí, Jaime ; Tormo, Mar ; Morado, Marta ; de la Iglesia, Silvia ; González-Rodríguez, Ana Pilar ; Bonanad, Santiago ; Melero, Virginia ; Torres, Williana ; Lavilla, Esperanza ; Benéitez, David ; Fernández, Ana ; Carnicero, Fernando ; on behalf of PETHEMA (Spain) ; Vidán, Julia ; González, Carmen ; Gaya, Anna ; Salido, Eduardo ; Sánchez, Piva ; Hernández, Inés

OBJECTIVES:

Terminal complement inhibitors can control intravascular hemolysis in Paroxysmal Nocturnal Hemoglobinuria (PNH) patients, although the hematological response achieved may not persist, and it may become unsatisfactory. The proximal (C3) complement inhibitor Pegcetacoplan (PEG) improves this situation in clinical trials, yet real-world data on its efficacy and safety are scarce.

METHODS:

This non-interventional, retrospective, multicenter study presents clinical and hematological data from the first PNH patients in Spain treated with PEG (n = 32, median 13 months, range 4-61).

RESULTS:

PEG rapidly increases the patient's hemoglobin (median increase of 2.65 g/dL in 4 weeks, > 2 g/dL increase in 56.3% of patients), reaching > 8, 10 and 12 g/dL in 93.7%, 87.5% and 46.9% of patients, respectively. Packed red blood cell transfusion requirements dropped from 62.5% to 15.6% of patients treated with PEG, and there was a fall in breakthrough hemolysis from 34.4% to 9.4% after 6 months of treatment. No thrombosis and only mild adverse events (n = 11 patients) were described with PEG therapy. Finally, physicians perceived important enhancements in patients' health-related quality of life.

DISCUSSION:

This real-world clinical data confirms that PEG produces significant and rapid improvements in hemoglobin and hemolytic parameters, with a favorable safety profile. The data extends that of earlier clinical trials and small real-world studies by offering a longitudinal (as opposed to cross-sectional) assessment of the patient responses across a large number of centers, following a variety of clinical practices..

CONCLUSION:

PEG treatment of patients with PNH produces significant and rapid improvements in hemoglobin and hemolytic parameters, with a favorable safety profile.

2026-12-31·Hematology

Switching patients with PNH from pegcetacoplan to iptacopan: a case series

Article

作者: Dingli, David ; Gurnari, Carmelo ; Maciejewski, Jaroslaw ; Sanikommu, Srinivasa ; Orland, Mark

OBJECTIVES:

Limited published data exist on switching therapy from pegcetacoplan to iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH).

METHODS:

Three patient cases were collected from the Mayo Clinic, Cleveland Clinic, and Levine Cancer Institute.

RESULTS:

Patient 1 is a 57-year-old woman with PNH. She experienced extravascular hemolysis (EVH) while on C5 inhibitors and breakthrough hemolysis (BTH) after switching to pegcetacoplan. Patient 2 is a 37-year-old woman seeking care for PNH, who was initiated on C5 inhibitor therapy and switched to pegcetacoplan. EVH and BTH were observed while she was on C5 inhibitor therapy, and transfusions were required with both C5 inhibitor and pegcetacoplan therapy. Patient 3 is a 58-year-old man with aplastic anemia and PNH who was initiated on C5 inhibitor therapy, then switched to pegcetacoplan. He experienced both EVH and BTH.

DISCUSSION:

All three patients switched from pegcetacoplan to iptacopan, and none experienced EVH or BTH while on iptacopan. After switching to iptacopan, all three patients improved hemoglobin levels and abating transfusion requirements.

CONCLUSION:

In this case series, three patients with PNH were successfully transitioned from pegcetacoplan to iptacopan monotherapy. No patients exhibited laboratory evidence of BTH, EVH, or intravascular hemolysis after switching to iptacopan.

731

项与 Pegcetacoplan 相关的新闻（医药）

2026-10-28

·Firstwordpharma

CEO says Biogen has 'returned to growth' as newer drugs, Apellis drive Q3 beat

Biogen said Wednesday that third-quarter revenues rose 11% year-over-year to $2.8 billion, beating analyst expectations of $2.6 billion, as its newer medicines, including those added through its $5.6-billion acquisition of Apellis Pharmaceuticals, helped offset declines in its legacy multiple sclerosis (MS) business."Our third-quarter results demonstrate that Biogen has returned to growth," stated CEO Chris Viehbacher. Seven of eight products in the pharma's growth portfolio grew by double digits, including spinal muscular atrophy drug Spinraza (nusinersen), which saw sales jump 23% to $461 million thanks to more patients converting to the high-dose regimen. Analysts were projecting $400 million.IQLIK outpacing IV launchRevenue for Eisai-partnered Alzheimer's treatment Leqembi increased 67% to $202 million – the Japanese drugmaker records global in-market sales of the drug – with $107 million of that in the US. The global figure edged past estimates of $199 million.Moreover, the Leqembi IQLIK subcutaneous formulation – approved for maintenance dosing last year and more recently as an at-home starting dose – has been outpacing Leqembi's original intravenous (IV) launch "by three and a half and [Eli Lilly's] Kisunla by six times," according to Alisha Alaimo, who heads Biogen North America operations, though she cautioned it was still early days.Alaimo noted that while the majority of IQLIK use comes from Leqembi IV prescription writers, Biogen is "now seeing writers who have never written an [anti-amyloid therapy] before, never wrote Kisunla, never wrote [Leqembi IV]. They're writing IQLIK – and we are also seeing physicians who have only ever written Kisunla write IQLIK." She called that "a dynamic change," especially in areas with long drive times, citing the West Coast as the fastest uptake given its geography and the "convenience factor."Apellis momentumMeanwhile, Biogen also reported the first full-quarter of sales for geographic atrophy treatment Syfovre and complement inhibitor Empaveli since the Apellis deal closed in May. Syfovre generated $191 million and Empaveli $52 million, for respective gains of 27% and 92% year-over-year."I think we've really realised a synergy between the folks from Apellis who've joined the Biogen teams," said Viehbacher on an analyst call Wednesday. "They've come together and when I look at Syfovre and Empaveli you can see 37% year-over-year growth of $243 million, that's already on a run rate of $1 billion."Overall, Biogen said the growth portfolio accounted for roughly two-thirds of its core pharmaceutical revenue. MS drug sales fell 13% in the quarter to $919 million.The company is keeping its full-year outlook for mid-single-digit revenue growth in 2026, but raised the lower end of its full-year adjusted earnings guidance by 20 cents, which now falls between $12.20 and $13 per share.Five key readoutsViehbacher also highlighted early-stage work as now being one of Biogen's key priorities, calling it "one of the most important and often most neglected areas of our industry." He admitted "we were not necessarily very good with that" — the company had gone three years without a single IND – but he says there has been "a radical overhaul of the whole research and early development area."The ambition is to have at least 20 programmes in early-stage development over the next few years. "This is a big shift in how we operate because Biogen historically has looked at things on an asset-by-asset basis," Viehbacher said.The company has five registrational study readouts on the horizon over the next year. Two Phase III trials of litifilimab, a IgG1 monoclonal antibody, are due in systemic lupus by year-end, with a third in cutaneous lupus to follow in the first half of 2027. That same half-year should also bring results from a Phase III study of felzartamab, a CD38-targeting antibody in development for antibody-mediated rejection (AMR) in kidney transplant recipients.Phase III results for zorevunersen, an antisense oligonucleotide it is co-developing with Stoke Therapeutics to treat Dravet syndrome, are expected towards the end of next year.

并购临床3期上市批准临床结果寡核苷酸

2026-10-28

·BioSpace

Biogen notches 3rd straight quarter of growth thanks to newer products, Apellis

iStock, z_wei Growth portfolio products like Leqembi and Spinraza helped drive Biogen to $2.8 billion in revenue for the third quarter. The bridge to growth that Biogen CEO Chris Viehbacher has been promising has officially reached the other side. The neuroscience specialist notched a third straight quarter of revenue growth, with $2.8 billion collected for the most recent period. Biogen in its third quarter earnings release pointed to the growth portfolio—which includes the Alzheimer’s disease therapy Leqembi and spinal muscular atrophy antisense oligonucleotide Spinraza—which jumped 49% year-over-year for $1.26 billion. Also contributing were new products from Apellis, which Biogen acquired in March for $5.6 billion. The third quarter marked the first full period of earnings from that acquisition. “Our third quarter results demonstrate that Biogen has returned to growth,” Viehbacher said in a prepared statement. Analysts had been expecting a slight beat of $2.6 billion on revenue and $2.14 earnings per share, which landed at $2.62, although recent acquired in-process research and development charges brought that down to a 14-cent loss when adjusted. “Overall, a solid quarter that reinforces confidence in execution and early Apellis integration,” Truist Securities wrote Wednesday morning. “That said, with earnings increasingly taking a back seat to the pipeline, we expect clinical catalysts to dominate BIIB shares over the next ~12-14 months.” Spinraza took in $461 million for the quarter, which Biogen attributed to accelerated conversions to a high-dose regimen that exceeded company expectations in all markets. Leqembi also continued its slow climb, adding $202 million, representing 67% growth year-over-year. Sales within the U.S. for the Alzheimer’s med were $107 million. Apellis’ eye injection Syfovre and complement inhibitor Empaveli combined for $243 million as compared to consensus of $216.8 million. Biogen slightly raised the lower end of its guidance to $12.20-$13, compared to $12-13 previously. Full year revenue is expected to land between $10.19-10.58 billion, Jefferies noted on Wednesday. The big biotech will host a conference call at 8:30 am ET.

并购寡核苷酸临床1期

2026-10-22

·医学进行时

AAO 2026视网膜亚专科日｜视网膜领域年度更新：8篇影响临床实践的文献、药物相关眼内炎症与系统用药视网膜毒性

AAO 2026 ·医学进行时编者按2026年10月9日，美国眼科学会（AAO）2026年会视网膜亚专科日在新奥尔良举行。"视网膜领域年度更新"单元由三位讲者完成，直接对应门诊和手术中的常见决策：Jason Hsu 教授梳理了过去一年中改变其临床实践的8篇文献，结论包括 AMD 高遗传风险人群通过生活方式干预可预防56%～60%的进展期 AMD、卒中或心肌梗死后无需暂停抗 VEGF 注射、增殖期糖尿病视网膜病变玻璃体切割术前4～7天注射抗 VEGF 药物的术后复发风险最低；Glenn J. Jaffe 教授总结了玻璃体腔注射药物相关眼内炎症的识别与处理；Jaclyn L. Kovach 教授更新了羟氯喹、戊聚糖多硫酸钠、胰高血糖素样肽-1受体激动剂及部分抗肿瘤药物的视网膜毒性与筛查建议。一、Jason Hsu：过去一年改变临床实践的8篇文献 Hsu 教授指出，大量同行评审论文难以及时进入一线医生的视野，他挑选的是过去一年中实际影响其诊疗习惯的研究，每页幻灯片均附 PubMed 链接二维码。1. 高遗传风险人群：生活方式仍能显著影响 AMD 进展 Seddon 等在 Ophthalmology 发表的前瞻性纵向研究纳入898只遗传风险评分处于最高三分位、基线为早期或中期年龄相关性黄斑变性（AMD）的眼，5年内23%进展为进展期 AMD。在从不吸烟者中，高风险生活方式（BMI≥25、热量摄入高于中位数、绿叶蔬菜<2.7份/周、鱼类<2份/周）者进展期 AMD 发生风险为理想生活方式者的3.3倍（HR 3.3，95% CI 1.8～6.4，P<0.001）；在曾吸烟者中为5.3倍（HR 5.3，95% CI 2.3～11.9，P<0.001）。人群归因风险估算显示，采取理想生活方式可预防56%（从不吸烟者）～60%（曾吸烟者）的进展期 AMD，对地图样萎缩（Geographic Atrophy，GA）的影响强于新生血管型。图1 高遗传风险人群中生活方式与进展期 AMD 的关联（Seddon JM, et al. Ophthalmology, 2026） Hsu 教授据此建议，对尚未进展的患者以及陪同就诊的进展期 AMD 患者家属，可以明确告知：控制体重、合理控制热量摄入、多吃鱼类和绿叶蔬菜，确实可以降低风险。2. AMD 患者饮食建议按分期细化 Keenan 等在 American Journal of Ophthalmology 发表的综述提出：地中海饮食适用于各期干性 AMD；中期 AMD 应重点强调鱼类摄入（每周2次及以上）；GA 患者应重点增加蔬菜和水果摄入，这与 GA 扩展减缓的关联最为明显。AREDS2 配方补充剂适用于中期及进展期 AMD，对中心凹外 GA 患者可延缓病变向中心凹扩展。Hsu 教授强调，AREDS2 补充剂与健康饮食是互补关系，不能互相替代。3. 卒中或心肌梗死后可继续抗 VEGF 治疗 Weng 团队在 JAMA Ophthalmology 发表的回顾性队列研究基于 TriNetX 电子病历网络，比较了心血管事件前14天至事件后6周内接受过抗血管内皮生长因子（Vascular Endothelial Growth Factor，VEGF）注射的患者，与事件前后3个月内均未注射的患者。倾向评分匹配后，卒中组1526对、心肌梗死组1523对。围事件期注射与死亡率或致残率升高无关；卒中后1年死亡风险 RR 0.52（95% CI 0.43～0.63），心肌梗死后1年死亡风险 RR 0.58（95% CI 0.49～0.68），阿柏西普、贝伐珠单抗、雷珠单抗之间无差异。Hsu 教授的结论是，近期发生卒中或心肌梗死的患者可以继续按计划注射。图2 卒中或心肌梗死围事件期抗 VEGF 注射与系统结局的森林图（Bordbar DD, et al. JAMA Ophthalmol, 2026）需要注意的是，该研究为观察性研究，围事件期注射组死亡风险更低的结果不宜解读为抗 VEGF 治疗具有心脑血管获益，其意义在于未发现安全性信号。4. 水溶性氯己定可替代聚维酮碘用于注射前消毒 Storey 团队在 Ophthalmology Retina 报告了单中心302,474次抗 VEGF 注射的数据：5%聚维酮碘组疑似注射后眼内炎发生率为0.022%（1/4529），0.05%水溶性氯己定组为0.014%（1/7057），P=0.34；培养阳性病例分别为10例和0例（P=0.25）。 Hsu 教授表示，考虑到部分患者对聚维酮碘刺激感耐受差，他已将较多患者改用水溶性氯己定，术后电话咨询和复诊明显减少。5. 糖尿病玻璃体切割术前抗 VEGF：4～7天为最佳间隔中山大学中山眼科中心赵秀娟、卢林团队在 Ophthalmology Retina 发表的竞争风险分析纳入608只因增殖期糖尿病视网膜病变（Proliferative Diabetic Retinopathy，PDR）行玻璃体切割术的眼，其中508只术前接受抗 VEGF 注射，按注射至手术间隔分为1～3天、4～7天、8～14天和>14天四组。以>14天为参照，4～7天组术后玻璃体积血或牵拉性视网膜脱离（Tractional Retinal Detachment，TRD）复发风险最低（HR 0.52，95% CI 0.30～0.88，P=0.015）；间隔超过14天者术后 TRD 风险最高。各组视力结局无显著差异。图3 PDR 玻璃体切割术前抗 VEGF 注射时机与术后复发风险（Zhan J, et al. Ophthalmol Retina, 2026） Hsu 教授据此将术前4～7天视为抗 VEGF 注射的最佳时间窗。6. 眼内气体配比错误：一项可以避免的医疗差错 Gaurav Shah 等在 Ophthalmology Retina 发表的短篇报告汇总了眼科医疗责任保险公司（OMIC）的12起因气体配比错误引发的诉讼，患者最终视力普遍很差，平均赔付金额接近50万美元。Hsu 教授据此在术中增加了"第二次暂停核对"：开灯后再配气，确认气体种类与浓度，由医生目视器械护士完成稀释，或由医生操作、护士监督；注射器内气体推至的位置有时可见弯月面，可作为二次核对依据。7. 第一眼视网膜裂孔，对侧眼格子样变性无需预防性激光 Hsu 团队在 Ophthalmology Retina 发表的回顾性队列研究纳入802例双眼格子样变性、首眼因视网膜裂孔接受治疗的患者，平均随访5.2年。对侧眼接受预防性激光组（83眼）与观察组（719眼）新发裂孔或孔源性视网膜脱离的复合终点发生率分别为24.1%和24.9%（P=0.87），孔源性视网膜脱离发生率分别为6%和5%（P=0.603）。他提醒，两组风险均不低，对侧眼仍需规律随访。图4 对侧眼预防性激光与观察组的视网膜脱离及裂孔累积发生曲线（Ni RL, et al. Ophthalmol Retina, 2026）8. "无滴眼液"玻璃体切割术加州大学圣迭戈分校 William R. Freeman 团队在 Retina 杂志报告：玻璃体切割术结束时玻璃体腔注射去溶剂曲安奈德（5 mg）和莫西沙星（500 μg），与术后常规局部用药的历史对照相比，前房炎症更轻、中央子野厚度更低、黄斑囊样水肿发生率更低（P<0.001），术后6个月最佳矫正视力提高20个字母（对照组8个字母，P=0.01）；两组眼压相近，均未发生眼内炎。Hsu 教授认为，这一方案有助于减轻患者负担和费用、提高满意度，值得尝试。二、Glenn J. Jaffe：药物相关眼内炎症的识别与处理 Jaffe 教授指出，随着生物制剂、基因治疗和小分子药物用于视网膜疾病，治疗相关眼内炎症越来越受到重视。这类炎症可能危及视力，也会影响医生和患者对药物的接受度。在分类上，药物相关眼内炎症可表现为前葡萄膜炎、中间葡萄膜炎、后葡萄膜炎或全葡萄膜炎，可伴或不伴视网膜血管炎及血管闭塞，需与内源性炎症、给药操作相关反应及感染性眼内炎鉴别。他特别指出，只有血管闭塞而没有炎症表现，不能诊断为血管炎。影像评估采用多模影像：超广角眼底照相与荧光素血管造影（Fluorescein Angiography，FA）优先，因为炎症体征常超出后极部；FA 显示渗漏但可能遮蔽血管，OCTA 显示血流但不显示渗漏，两者互补。OCT 上的玻璃体或视网膜内高反射点可能是炎症的早期征象。Jaffe 教授所在的阅片中心已建立标准化的预设影像变量，用于药物上市后监测和临床试验评估。不同药物的炎症表现有一定规律。布西珠单抗（brolucizumab）相关炎症多在数次注射、数月后出现，后段炎症前可先有前房或玻璃体细胞，可出现局灶性视网膜变白、血管炎或血管闭塞。pegcetacoplan 相关炎症多在首次注射后发生，较为罕见，临床试验中未观察到，上市后才被报告，可表现为类似万古霉素相关出血性闭塞性视网膜血管炎（Hemorrhagic Occlusive Retinal Vasculitis，HORV）的闭塞性血管炎，可导致视力丧失。对法瑞西单抗在糖尿病黄斑水肿和 nAMD 关键试验中的眼内炎症事件进行盲法、多模影像标准化复核后，未发现固定模式，也未见血管炎或闭塞性血管炎，上市后有少数病例报告。图5 布西珠单抗相关眼内炎症的典型特征治疗按解剖部位选择：前节炎症强化局部糖皮质激素；玻璃体炎症或急性血管炎使用全身或局部糖皮质激素；转为慢性时可考虑抗代谢药或生物制剂等免疫治疗。怀疑药物相关炎症时应停用该药，Jaffe 教授不建议再次激发试验，应换用其他药物；注射前对可疑病例应先做仔细的裂隙灯检查。他认为未来需要统一的报告标准和治疗路径，对即将上市的基因治疗等新药做好炎症谱特征描述，并开发经过验证的眼内炎症与葡萄膜炎生活质量量表。三、Jaclyn L. Kovach：系统用药相关视网膜毒性更新羟氯喹：OCT 成为主要筛查工具 AAO 于2025年发布羟氯喹视网膜病变筛查建议修订版。Kovach 教授强调的要点包括：OCT 联合广角眼底自发荧光（FAF）为主要筛查手段，视野和多焦视网膜电图为确认性检查；24-2C 等新视野程序可同时筛查中心凹旁和中心周围区域，这一点很重要，因为欧洲裔患者多表现为中心凹旁型损害，东亚裔患者多表现为中心周围型损害。推荐剂量仍为≤5.0 mg/kg/d（按实际体重）。除剂量和疗程外，合并肾病、使用他莫昔芬、高龄起始用药，以及 AMD 等影响筛查判读的黄斑疾病，均为需要关注的危险因素。图6 AAO 羟氯喹视网膜病变筛查建议（2025修订版）要点戊聚糖多硫酸钠：累积剂量是最主要危险因素自2018年首个病例系列报告以来，长期使用戊聚糖多硫酸钠与特征性色素性黄斑病变的关联已得到确认。累积剂量是最主要的危险因素，病理改变为视网膜色素上皮和光感受器损伤以及内层脉络膜缺血。FAF 上最具特征的表现为双眼对称、融合的高低自发荧光斑点，早期局限于血管弓内，晚期可进展为中心萎缩并超出血管弓。开始用药时应完成眼底照相、FAF 和 OCT 基线检查，此后每年筛查；一旦出现视网膜病变，应与处方医生和患者讨论停药或减量。与羟氯喹相同，停药后病变不可逆，并可能继续进展。GLP-1 受体激动剂：AMD 方面未见风险增加，DR 方面证据尚不一致美国约每8名成人中有1人使用过胰高血糖素样肽-1受体激动剂（Glucagon-Like Peptide-1 Receptor Agonist，GLP-1 RA），约一半成人符合减重用药条件。多项回顾性队列研究显示，GLP-1 RA 可能与非新生血管性 AMD 风险降低相关，未见新生血管性 AMD 风险增加。与糖尿病视网膜病变的关系尚不明确，大型数据库研究结论不一，需要前瞻性研究验证。目前医学界的共识是，优先考虑这类药物在降低心脑血管风险方面的获益。所有糖尿病患者均应进行基线眼科检查和定期随访；血糖控制快速改善的患者可缩短随访间隔，已有 PDR 者可考虑在用药前先行治疗。图7 GLP-1 RA 与 AMD 关联的研究汇总抗肿瘤药物：识别特征性表现紫杉醇和多西他赛等紫杉烷类微管抑制剂可引起荧光素血管造影无渗漏的黄斑囊样水肿，停药后通常消退。MEK 抑制剂可引起视网膜下液积聚，与中心性浆液性脉络膜视网膜病变的区别在于：多为多灶性积液，脉络膜厚度正常，且不伴色素上皮脱离。免疫检查点抑制剂偶可引起葡萄膜炎和 Vogt-小柳-原田样综合征。 Kovach 教授总结，眼科医生需要了解可能引起新型视网膜毒性的药物及其筛查方案，详细询问用药史是前提；部分药物所致损害不可逆，需要生物标志物改善风险分层。在讨论环节，她补充：上述药物如有条件，均建议在用药前完成基线检查，使用 GLP-1 RA 的糖尿病患者尤其需要。小结本单元的内容几乎都能直接用于日常诊疗：给 AMD 高风险人群的生活方式与饮食建议、心脑血管事件后抗 VEGF 治疗的连续性、玻璃体切割术前注射时机，以及羟氯喹筛查方案的更新。其中关于 PDR 术前抗 VEGF 注射时机的研究来自中山眼科中心，是本单元唯一入选的中国团队研究。参考文献： 1. Seddon JM, De D, Rosner B. Quantifying Effects of Diet and Lifestyle Changes on Progression to Advanced Age Related Macular Degeneration in High Genetic Risk Individuals. Ophthalmology. 2026;133(3):333-349. doi:10.1016/j.ophtha.2025.09.030 2. Keenan TDL, Hébert M, Chew EY, Johnson MW. What Should Patients With Age-Related Macular Degeneration Eat? Am J Ophthalmol. 2026;288:12-21. doi:10.1016/j.ajo.2026.04.003 3. Bordbar DD, et al. Peri-Event Intravitreal Anti-VEGF and Systemic Outcomes After Stroke or Myocardial Infarction. JAMA Ophthalmol. 2026;144(5):415-422. doi:10.1001/jamaophthalmol.2026.0460 4. Bates B, et al. Endophthalmitis Incidence after Intravitreal Injection of Anti-VEGF Agents with Povidone-Iodine versus Aqueous Chlorhexidine Antisepsis. Ophthalmol Retina. 2025;9(10):928-933. doi:10.1016/j.oret.2025.03.022 5. Zhan J, et al. Timing of Preoperative Anti-VEGF for Vitrectomy in Proliferative Diabetic Retinopathy: A Competing-Risks Analysis of a Large Cohort. Ophthalmol Retina. 2026;10(5):532-542. doi:10.1016/j.oret.2025.12.019 6. Ni RL, et al. Efficacy of Prophylactic Laser Treatment of Lattice Degeneration in the Fellow Eyes of Patients with Retinal Breaks in the First Eye. Ophthalmol Retina. 2026. doi:10.1016/j.oret.2026.06.016 7. Ali AL, et al. Dropless Pars Plana Vitrectomy Using Intravitreal Decanted Triamcinolone and Moxifloxacin: Safety and Efficacy. Retina. 2026. doi:10.1097/IAE.0000000000004947 8. Marmor MF, et al. Special AAO Report: Recommendations on Screening for Hydroxychloroquine Retinopathy (2025 Revision). Ophthalmology. 2026;133(4):439-450. doi:10.1016/j.ophtha.2025.11.001 9. 待核实：Gaurav Shah 等关于眼内气体配比错误诉讼的 Ophthalmology Retina 短篇报告（标题、年份） END 更多会议信息&学术资讯，敬请关注：“医学进行时”微信公众号

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

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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

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适应症	最高研发状态	国家/地区	公司	日期
肾小球疾病	临床3期	巴西	Swedish Orphan Biovitrum AB	2027-01-21
肾小球疾病	临床3期	加拿大	Swedish Orphan Biovitrum AB	2027-01-21
肾小球疾病	临床3期	芬兰	Swedish Orphan Biovitrum AB	2027-01-21
肾小球疾病	临床3期	法国	Swedish Orphan Biovitrum AB	2027-01-21
肾小球疾病	临床3期	德国	Swedish Orphan Biovitrum AB	2027-01-21
肾小球疾病	临床3期	意大利	Swedish Orphan Biovitrum AB	2027-01-21
肾小球疾病	临床3期	沙特阿拉伯	Swedish Orphan Biovitrum AB	2027-01-21
肾小球疾病	临床3期	西班牙	Swedish Orphan Biovitrum AB	2027-01-21
肾小球疾病	临床3期	瑞士	Swedish Orphan Biovitrum AB	2027-01-21
肾小球疾病	临床3期	英国	Swedish Orphan Biovitrum AB	2027-01-21

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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 (GALE \| OAKS, DERBY, and GALE, CTgov)	临床3期	年龄相关性黄斑变性 \| 地图样萎缩 \| II型糖原贮积病	792	PEGCETACOPLAN (APL-2) (EOM: Pegcetacoplan/Pegcetacoplan)	範製範窪餘憲築膚遞構 = 齋鏇觸顧蓋選鬱醖範壓構膚獵餘獵選壓網繭夢 (餘廠獵簾齋壓夢壓網窪, 淵簾觸選夢選鏇選願製 ~ 醖膚醖觸壓願蓋網獵艱) 更多	-	2026-10-28
NCT04770545 (GALE \| OAKS, DERBY, and GALE, CTgov)	临床3期	年龄相关性黄斑变性 \| 地图样萎缩 \| II型糖原贮积病	792	PEGCETACOPLAN (APL-2) (Monthly: Sham/Pegcetacoplan)	範製範窪餘憲築膚遞構 = 廠願鏇製醖網憲鹹壓積構膚獵餘獵選壓網繭夢 (餘廠獵簾齋壓夢壓網窪, 繭淵積鑰夢壓窪築夢鹽 ~ 襯壓鏇觸鹽膚遞鹹艱繭) 更多	-	2026-10-28
PRISM-C3 (ASN2026)	临床4期	膜增生性肾小球肾炎 \| C3肾小球病	100	Pegcetacoplan	壓壓廠夢醖壓遞鹹廠製(獵艱築憲衊糧鏇鬱獵艱) = 鏇艱觸顧積齋構構鬱憲醖構鏇壓獵艱築淵餘網 (衊積鏇淵願觸憲蓋糧獵 )	积极	2026-10-24
NCT05067127 (VALIANT, ASN2026)	临床3期	膜增生性肾小球肾炎 \| C3肾小球病	120	Pegcetacoplan	齋築壓廠壓簾築繭鹹夢(鑰觸觸鹽鏇顧獵選築範) = 觸憲製鑰鹹蓋鑰艱遞顧襯範鬱糧衊膚鹹遞糧鹽 (壓鏇糧鏇壓蓋選範膚鏇, -6.6 ~ 3.9) 更多	积极	2026-10-24
NCT05067127 (VALIANT, ASN2026)	临床3期	膜增生性肾小球肾炎 \| C3肾小球病	120	Pegcetacoplan (IFTA ≤25%)	齋築壓廠壓簾築繭鹹夢(鑰觸觸鹽鏇顧獵選築範) = 淵製鹽鏇積獵襯鏇窪鹹襯範鬱糧衊膚鹹遞糧鹽 (壓鏇糧鏇壓蓋選範膚鏇, -3.9 ~ 7.3) 更多	积极	2026-10-24
ASN2026	临床3期	阵发性睡眠性血红蛋白尿症	132	Pegcetacoplan 1,080 mg	築艱網醖齋憲獵艱糧窪(顧顧遞獵夢鏇窪廠選選) = 鹽顧膚衊鬱蓋餘餘構鹹鹽選範顧蓋衊鹹艱壓鬱 (壓鏇淵憲艱觸製憲鑰夢 ) 更多	积极	2026-10-23
PRESERVE-C3Tx (ASN2026)	临床3期	膜增生性肾小球肾炎 \| C3肾小球病	30	Pegcetacoplan	蓋鏇觸簾襯簾淵蓋積淵(築簾構膚觸獵窪鹽願蓋) = The most common adverse events (AEs) were infections and were similar to those seen in the general population following kidney transplantation. The rate of serious infections was higher in the first 6 months post-transplantation, but similar between 6 and 24 months post-transplantation and to that reported in the general kidney transplant population. 齋選淵廠膚構鹽鏇繭鹽 (鏇鑰築艱觸壓醖憲鬱範 )	积极	2026-10-22
NCT05067127 (VALIANT, ASN2026)	临床3期	膜增生性肾小球肾炎 \| C3肾小球病	53	Pegcetacoplan	製選膚範簾簾襯淵築蓋(蓋範餘遞築鹽鏇憲築衊) = 窪糧憲簾廠夢願蓋範願顧選醖鏇淵製憲鬱鹹膚 (積壓願窪齋繭簾顧遞餘, -77.0 ~ -54.1) 更多	积极	2026-10-22
ISTH2026	N/A	阵发性睡眠性血红蛋白尿症	240	Proximal complement inhibitors	製壓醖艱壓觸鹹鹽膚顧(鏇鏇壓壓衊衊襯鹹顧餘) = 簾醖遞繭觸蓋憲範齋製鏇蓋簾網鬱網醖獵鑰夢 (範淵壓繭糧構鹹蓋製網 ) 更多	积极	2026-07-13
ISTH2026	N/A	阵发性睡眠性血红蛋白尿症	240	C5 inhibitors	-	积极	2026-07-13
NCT05067127 \| NCT05809531 (VALIANT, Pubmed \| Clin J Am Soc Nephrol)	临床3期	膜增生性肾小球肾炎 \| C3肾小球病	55	Pegcetacoplan	願餘觸醖壓艱積醖鬱製(鹹鹹顧糧鑰艱窪壓鹽壓): P-Value = 0.002 更多	积极	2026-05-14
	临床3期	膜增生性肾小球肾炎 \| C3肾小球病	55	Placebo	願餘觸醖壓艱積醖鬱製(鹹鹹顧糧鑰艱窪壓鹽壓): P-Value = 0.002 更多	积极	2026-05-14
EHA2026	N/A	阵发性睡眠性血红蛋白尿症	10	EculizumabDanicopanRavulizumabPegcetacoplan + EculizumabDanicopanRavulizumabPegcetacoplan + EculizumabDanicopanRavulizumabPegcetacoplan + EculizumabDanicopanRavulizumabPegcetacoplan	鑰積餘顧廠廠餘蓋鹽壓(醖襯簾鑰壓鏇鑰糧範獵) = During a median follow-up of 49.5 months, 3/10 patients required therapy modification due to persistent anemia compatible with EVH. 觸膚醖獵遞餘範積製憲 (憲襯鑰醖願艱網範衊鹽 )	积极	2026-05-12
EHA2026	N/A	阵发性睡眠性血红蛋白尿症	38	Pegcetacoplan	遞製簾願膚醖願網齋願(繭選膚簾繭襯鹹餘醖壓) = 鏇獵蓋憲製醖築繭糧鹹廠蓋壓獵獵齋繭製顧壓 (鏇糧鹹淵製淵積壓簾顧 ) 更多	积极	2026-05-12