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更新于：2026-11-06

Pegcetacoplan

更新于：2026-11-06

概要

概要

基本信息

药物类型

合成多肽、环肽药物

别名

Aspaveli/Empaveli、Pegcetacoplan (USAN/INN)、APL 2

+ [6]

靶点

作用方式

抑制剂

作用机制

C3抑制剂(补体C3抑制剂)

治疗领域

遗传病与畸形眼部疾病血液及淋巴系统疾病+ [7]

在研适应症

C3肾小球病膜增生性肾小球肾炎地图样萎缩+ [15]

非在研适应症

肌萎缩侧索硬化冷凝集素病免疫球蛋白a肾病+ [6]

原研机构

Apellis Pharmaceuticals, Inc.

在研机构

Apellis Pharmaceuticals, Inc.

Swedish Orphan Biovitrum AB

Handok, Inc.

+ [3]

非在研机构-

权益机构

SFJ Pharmaceuticals, Inc.

Biogen, Inc.

Swedish Orphan Biovitrum Ltd.

+ [5]

最高研发阶段批准上市

首次获批日期

美国 (2021-05-14),

阵发性睡眠性血红蛋白尿症

最高研发阶段(中国)-

特殊审评快速通道 (美国)、孤儿药 (美国)、孤儿药 (欧盟)、孤儿药 (日本)、孤儿药 (澳大利亚)、优先审评 (澳大利亚)、优先审评 (美国)、孤儿药 (英国)

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结构/序列

Sequence Code 550935032

来源: *****

关联

项与 Pegcetacoplan 相关的临床试验

NCT07898774

/ Not yet recruiting临床3期

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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246

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 ; Tormo, Mar ; Martí, Jaime ; de la Iglesia, Silvia ; González-Rodríguez, Ana Pilar ; Morado, Marta ; 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.

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项与 Pegcetacoplan 相关的新闻（医药）

2026-11-02

·腾讯网

从足细胞生物学走向肾小球医学——第15届国际足细胞大会精要汇总

编者按近期，第15届国际足细胞大会（15th International Podocyte Conference）核心会议内容在线发表于Kidney International。此次会议于2025年6月10~13日在德国汉堡举办，同时宣告国际肾小球疾病学会（International Society of Glomerular Disease，ISGD）正式成立，标志着肾脏病研究从单纯足细胞生物学拓展为“肾小球医学”这一整合化学科。会议汇集500余名线上线下参会学者，收到170余篇摘要，围绕遗传调控、多组学与系统医学、各类肾小球疾病发病机制、新型生物标志物与转化治疗展开研讨。本文基于该会议报告，梳理肾小球疾病领域前沿突破，分析现存挑战，展望精准肾小球医学发展方向。十余年前第10届国际足细胞大会就已经开启研究范式转变：研究边界不再局限足细胞本身，而是延伸到肾小球整体细胞、基质微环境，催生了肾小球医学的学科概念。传统肾脏病多以疾病表型驱动研究，而当前领域倡导转向以肾小球健康为核心的研究模式，结合人群遗传学、单细胞空间转录组、冷冻电镜结构生物学、动物模型推进机制解析，同时关注患者真实体验、专科可及性与临床试验设计优化，弥合基础发现与临床治疗之间的鸿沟。1、遗传与基因调控研究新进展大规模全基因组关联研究（GWAS）极大拓展蛋白尿相关遗传图谱。对超过80万人群的分析，鉴定出135个与尿白蛋白肌酐比值（UACR）相关的遗传位点，位点数量较既往增加一倍以上。将遗传关联结果与肾脏转录组、蛋白质组联合分析，可优先筛选致病基因，例如FOXD2，揭示参与足细胞稳态与肾病易感性的调控通路，体现人群遗传学与临床遗传学互补价值，为慢性肾脏病、蛋白尿疾病的基因挖掘提供新思路（图1）。从遗传发现走向临床转化的典型代表是APOL1变异，该变异与局灶节段性肾小球硬化（FSGS）密切相关，基于该靶点已经开展多项靶向临床试验。多基因风险评分、多层级基因优先排序工具逐步用于患者分层，预测肾病进展，支撑精准医疗实施。跨物种模型继续贡献机制线索，黑腹果蝇肾细胞可用于机械感知与功能基因筛选，cheerio、piezo、Duf(Neph1)等基因在哺乳动物足细胞中存在功能保守性，为高通量基因功能验证提供简便体系。图1. 遗传与基因调控研究新进展2、蛋白质组、代谢组与足细胞应激机制足细胞是肾小球代谢调控、蛋白稳态与线粒体适应的核心细胞。同位素示踪、微流控等离体与在体代谢图谱技术证实，足细胞拥有区别于肾脏其他区段独特的氨基酸代谢通路，积极参与营养转运与利用；同时证实肠-肾轴存在，肠道来源代谢物显著影响肾小球代谢状态，参与慢性肾脏病进程（图2）。线粒体生物学研究揭示足细胞线粒体呼吸能力高，线粒体功能存在显著性别差异；线粒体与溶酶体协同维持足细胞能量平衡与细胞完整性，在足细胞衰老过程中扮演关键角色。蛋白酶体是足细胞主要蛋白降解系统；蛋白酶体损伤会诱导免疫蛋白酶体激活，通过去泛素化酶UCH-L1氧化修饰加重足细胞损伤，形成蛋白毒性应激正反馈环路。会议重点报道exopher（外泌体）：足细胞一种适应性应答方式，通过大型细胞外囊泡排出受损蛋白与免疫复合物，该现象在膜性肾病尤为突出；尿液中释放抗原-抗体复合物，有望成为反映疾病活动度的无创生物标志物。图2. 蛋白质组、代谢组与足细胞应激机制研究进展3、系统医学与疾病模型：单细胞、空间组学赋能肾小球研究系统医学、单细胞与计算生物学重塑肾小球免疫微环境的认知。子痫前期被视作生理应激下足细胞适应的经典模型：妊娠状态下足细胞数量与滤过需求不匹配会诱发肾脏损伤；壁上皮细胞雌激素信号改变，造成足细胞适应障碍，诱发子痫样损伤，不仅影响母体肾脏，还会降低子代肾单位储备，提示性别差异相关肾病易感性起源与肾脏祖细胞功能密切相关（图3）。IgA肾病研究借助多组学与基因调控网络解析疾病相关细胞状态；基于单细胞RNA测序建立足细胞损伤评分，可量化FSGS模型中足细胞损伤程度，与白蛋白尿水平相关，同时发现黏附程序丢失、昼夜节律紊乱与足细胞损伤双向关联。针对超过200例糖尿病肾病患者、合计500万细胞的高分辨率空间转录组研究，分析30万以上免疫细胞，发现部分患者肾小球内存在高度组织化B细胞集群，依赖CXCL13/BAFF信号，发生局部克隆扩增、分化为浆细胞；存在该B细胞微环境的患者更快进展至终末期肾病。空间定义的免疫微环境，为患者分层、免疫靶向治疗提供全新依据。图3. 系统医学与疾病模型研究进展4、各类肾小球疾病机制与转化突破01IgA肾病IgA肾病“多重打击”模型仍是理解发病核心：半乳糖缺陷型IgA1（Gd-IgA1）过度生成、自身抗体产生、免疫复合物沉积驱动肾小球炎症。纳入>10 000例IgA肾病病例、近30 000例对照的大规模遗传研究鉴定33个全基因组显著风险位点，多数集中免疫调控、黏膜免疫、细胞因子信号通路。针对数量性状的遗传分析找到调控IgA总量、缺陷IgA1的基因位点，进一步夯实发病机制（图4）。药物转化方面，双重内皮素-血管紧张素受体拮抗剂Sparsentan的临床试验显示，可显著降低蛋白尿，同时保护肾功能，支持其用于IgA肾病管理。领域共识强调整合遗传风险、生物标志物、组织学信息，实现IgA肾病的患者分层与个体化治疗，推动机制导向的精准诊疗。图4. IgA肾病研究进展 02微小病变肾病（MCD）与FSGS传统观点认为蛋白尿来自滤过电荷选择性丢失或者肾小管重吸收缺陷，新的生物物理模型提出：裂孔隔膜缩短发生在白蛋白尿出现之前，和蛋白尿严重程度密切相关；足细胞损伤是连续动态过程，而不是单纯足突融合的二元状态。提出凝胶压缩模型：足细胞提供支撑力维持肾小球基底膜（GBM）压缩；支撑力丢失造成基底膜解压，白蛋白通透性升高。蛋白尿分为早期裂孔依赖阶段、后期分流依赖进展阶段（图5）。冷冻电子断层扫描看到天然裂孔隔膜呈现渔网样有序结构；裂孔隔膜不只是静态屏障，更是动态信号平台，nephrin、Neph1、podocin构成复杂互作蛋白网络。大会重点报道免疫介导亚型：抗nephrin自身抗体是激素敏感肾病综合征、MCD的致病驱动因子。抗体阳性患者激素应答更好；频繁复发患者利妥昔单抗疗效佳；抗体水平还可以预测移植后复发风险，是极具前景的诊断、预后标志物，亟待开发可广泛应用的检测手段，指导个体化免疫抑制策略。图5. FSGS研究进展 03GBM病与膜性肾病肾小球基底膜不是静态支架，而是动态重构结构，包含超过160种组分；α3α4α5(IV)胶原网络对机械稳定性至关重要，存在昼夜节律性周转与应激适应性重塑。Alport综合征仍然存在大量漏诊，基因检测尚未常规应用；亟需更加敏感的替代终点，加速新药研发（图6）。膜性肾病作为足细胞靶向抗体驱动的自身免疫疾病，靶抗原以PLA2R、THSD7A为主，激活补体通路。补体靶向临床试验结果异质性大；B细胞耗竭治疗已经写入KDIGO指南。Daratumumab、CAR-T、双特异性抗体是难治膜性肾病下一代候选方案。图6. GBM病与膜性肾病研究进展 04补体介导肾小球疾病（C3肾小球肾炎）旁路补体通路过度激活是C3肾小球病核心致病机制。依库珠单抗、avacopan、iptacopan、pegcetacoplan等补体抑制剂临床试验证实可以降低蛋白尿、稳定肾功能，但个体应答差异明显。英国罕见肾病登记队列数据显示持续蛋白尿患者进展至肾衰风险极高（图7）。图7. C3肾小球肾炎和补体介导肾小球疾病研究进展 05ANCA血管炎、狼疮肾炎与壁上皮细胞（PEC）新月体肾炎中T细胞亚群（Th1、Th17、GranzymeK⁺CD8⁺T细胞）介导组织损伤，为选择性免疫调节提供靶点。壁上皮细胞EGFR信号激活驱动新月体形成，抑制该通路可减轻大动物模型肾脏病变（图8）。REGENCY临床试验证实奥妥珠单抗（Obinutuzumab）可提升狼疮肾炎完全缓解率，实现深度B细胞清除，减少复发。重复肾活检、尿液生物标志物、分子图谱，助力狼疮肾炎个体化监测。图8. ANCA血管炎、狼疮肾炎与壁上皮细胞（PEC）研究进展5、奖项与未来展望此次国际足细胞大会期间，ISGD颁发两项重要奖项：Jochen Reiser获得MarilynG.Farquhar终身成就奖，表彰其在足细胞生物学领域和肾小球疾病研究方面的重大发现；Nicola Tomas获得Wilhelm Kriz中青年研究者成就奖，表彰其在膜性肾病新抗原发现方面的贡献。未来肾小球医学的挑战仍十分突出：免疫细胞功能解析、跨人群生物标志物验证、区分足细胞与壁上皮细胞的贡献、优化靶向治疗方案。优先发展方向包括多组学深度整合、比较模式生物平台、稳健的生物标志物验证、适应性临床试验、早期干预策略。未来肾小球疾病卓越中心将促进全球协作，加快从基础走向临床。此外，会议公布后续会议安排：2026年美国加尔维斯顿举办ISGD临床峰会；2027年第16届国际足细胞大会在美国安娜堡举办。参考文献Khbouz B, et al. Kidney International. 2026; https://doi.org/10.1016/j.kint.2026.07.026.（来源：《肾医线》编辑部）声明：本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。

2026-10-31

·抗体君

抉择: 高浓度小体积 vs. 低浓度大体积

抗体君感恩您的一路相伴 Let's start learning now 做事，做好事，事做好，好做事 Q9921 高浓度小体积与低浓度大体积如何选？选择高浓度低体积还是低浓度高体积，核心在于平衡制剂可行性、患者可及性与开发风险。传统皮下给药受限于自动注射笔（≤2.25 mL）和预灌封注射器（≤15 mL但需手动推注），迫使开发者追求100-200 mg/mL的超高浓度，但这会带来三大难题：物理不稳定性（聚集、颗粒）、高粘度（推注困难、需粗针头）、生产工艺挑战（过滤、灌装）。而新型贴敷式给药装置（OBDS）支持3.5-25 mL单次给药，使得中低浓度（30-70 mg/mL）、大体积策略成为可行替代。决策建议：对于中等剂量（如每月240 mg），使用30 mg/mL浓度仅需8 mL，完全在OBDS能力内，可规避高浓度风险。对于超高剂量（如每月2400 mg），即使100 mg/mL仍需24 mL，高浓度开发仍不可避免。因此，早期应评估目标剂量落在剂量-体积矩阵的哪个区域，选择风险更低的路径。 ♀抗体君♀ Q9922 皮下给药为何受体积和浓度双重束缚？皮下给药的体积和浓度束缚源于解剖生理限制与设备能力的共同作用。解剖上，皮下组织弹性有限，快速注入超过1-2 mL液体会引起疼痛、肿胀甚至药液渗漏。传统设备进一步强化了这一限制：自动注射笔设计上限约2.25 mL，预灌封注射器虽可达15 mL但需医护人员手动推注，且高粘度药液会导致推注力过大（尤其对关节炎患者不友好）。为了实现与静脉输注（100-250 mL，浓度<30 mg/mL）相当的疗效，开发者被迫在有限体积内浓缩足够药量，形成“高浓度-高粘度-粗针头-高疼痛”的恶性循环。此外，高浓度还带来聚集和免疫原性风险。然而，新型OBDS的出现正在打破这一束缚——它支持5-25 mL大体积、可隐藏针头、恒速给药，使得开发者可以“用体积换浓度”，选择更稳健的中低浓度制剂路径，避免攀登超高浓度的技术高峰。 ♀抗体君♀ Q9923 静脉与皮下给药的核心利弊对比？静脉输注（IV）与皮下注射（SC）在多个维度上存在显著差异。IV优势：100%生物利用度、可容纳大体积（100-250 mL）低浓度（<30 mg/mL）药液、制剂开发难度低。IV劣势：需医疗机构完成、耗时30分钟至数小时、占用输液椅资源、患者频繁往返、血药浓度峰值高可能增加副作用风险。 SC优势：患者可在家自行给药（提升依从性和生活质量）、释放医疗资源、给药仅需数分钟、血药浓度平缓可能降低输液反应风险。SC劣势：生物利用度通常低于100%且个体差异大、受体积限制被迫开发高浓度制剂（100-200 mg/mL）、面临聚集/粘度/生产挑战。关键权衡点：SC虽便利，但若开发失败率高或需更高剂量补偿低生物利用度，可能得不偿失。临床数据显示，部分药物SC与IV疗效等效，且输液反应率更低（如达雷妥尤单抗）。选择时需综合药物分子性质、目标剂量、患者人群和开发预算。 ♀抗体君♀ Q9924 高浓度制剂面临哪三座大山？高浓度制剂开发面临物理不稳定性、高粘度和免疫原性三大核心挑战。第一座大山：物理不稳定性。高浓度下蛋白质分子间距缩小，相互作用急剧增强，导致可逆自关联（二聚体、寡聚体形成）和不可逆聚集（亚可见/可见颗粒）。聚集不仅影响药效，更可引发免疫原性。生产端表现为过滤膜堵塞、灌装困难、精度下降。应对策略包括添加蔗糖/海藻糖等稳定剂、聚山梨酯类表面活性剂、精氨酸等氨基酸，或采用冻干工艺，但无法完全消除风险。第二座大山：高粘度。理想注射粘度应低于10 cP，而高浓度制剂（如200 mg/mL mAb）粘度可达20-50 cP。高粘度导致：患者推注困难（尤其老年/关节炎患者）、需使用更粗针头增加疼痛、生产工艺（超滤/灌装）效率下降第三座大山：免疫原性。蛋白质聚集体是抗药抗体（ADA）产生的关键风险因素，ADA可导致药物清除加快、疗效下降甚至严重过敏。虽然临床数据显示SC与IV的ADA发生率相似（如阿巴西普、托珠单抗），但避免聚集始终是降低免疫原性风险的核心目标。 ♀抗体君♀ Q9925 低浓度大体积制剂的开发优势有哪些？低浓度大体积制剂利用OBDS（3.5-25 mL）等设备，展现出显著优势。优势一：规避超高浓度挑战。OBDS允许单次注射5-25 mL，意味着开发者可选用30-70 mg/mL的中低浓度，避免攀登100-200 mg/mL的“浓度高峰”。例如Ravulizumab的SC制剂采用70 mg/mL（其IV制剂为100 mg/mL），通过两个3.5 mL OBDS给药；若使用更大容量OBDS，浓度还可进一步降低。优势二：无需渗透促进剂。透明质酸酶（rHuPH20）虽能促进扩散，但存在相容性差、增加制剂复杂性、本身导致高粘度等问题。大体积OBDS可在不依赖rHuPH20的情况下实现有效给药，简化配方、降低开发风险。优势三：显著降低开发难度。中低浓度制剂的物理稳定性更好、聚集风险更低、粘度易于控制、生产工艺更成熟（接近IV制剂），直接转化为更短的开发周期、更低的成本和更高的成功率。优势四：更好的患者体验。低粘度可使用更细针头，配合OBDS隐藏针头设计，显著减轻疼痛和针头恐惧。 ♀抗体君♀ Q9926 OBDS如何解锁皮下给药的体积限制？贴敷式给药装置（OBDS）通过填补自动注射笔与输液泵之间的容量空白，革命性地解锁了皮下给药的体积限制。传统自动注射笔容量≤2.25 mL，是高浓度需求的根源；预灌封注射器虽可达15 mL但需手动推注，对高粘度药液不友好；输液泵容量虽大（≥50 mL）但体积庞大、需管路连接，限制患者活动。OBDS的容量范围3.5-25 mL完美覆盖了临床常用剂量区间。其核心技术特点：贴敷式设计，可贴于手臂或腹部，解放双手；隐藏针头，缓解针头恐惧症；恒速/恒压给药，避免手动推注不稳定带来的疼痛，注射时间可调（数分钟至数十分钟）；一次性使用，无需清洗灭菌。临床实例：ULTOMIRIS®使用两个3.5 mL OBDS（总7 mL），EMPAVELI®使用单个20 mL OBDS。OBDS使开发者得以灵活选择浓度-体积组合——对于每月240 mg的中等剂量，30 mg/mL仅需8 mL，完全在OBDS能力内，无需开发100 mg/mL超高浓度制剂。 ♀抗体君♀ Q9927 剂量-体积-浓度三维平衡如何决策？剂量-体积-浓度的三维平衡可通过矩阵工具进行决策。以80 kg患者、每月给药为例：总剂量（mg）= 剂量（mg/kg/月）× 80。所需体积（mL）= 总剂量 ÷ 浓度（mg/mL）。绿色区域（单次OBDS可行）：如每月3 mg/kg（240 mg），使用30 mg/mL仅需8 mL，处于OBDS舒适区。对于此类中等剂量，首选低浓度大体积策略，规避高浓度风险。黄色区域（需稀释或多设备）：如每月10 mg/kg（800 mg），30 mg/mL需26.7 mL，超过单OBDS能力；100 mg/mL需8 mL可行。需权衡：是开发100 mg/mL高浓度，还是接受两个OBDS或多个注射。橙色/红色区域（高难度）：如每月30 mg/kg（2400 mg），100 mg/mL仍需24 mL，可能需要多个OBDS或输液泵。对于超高剂量，高浓度开发仍不可避免。决策原则：早期评估目标剂量落在哪个区域；优先选择绿色路径；若落入黄色区域，评估高浓度开发的技术风险与多设备给药的依从性影响；红色区域需提前布局高浓度制剂和复杂给药方案的协同开发。 ♀抗体君♀ Q9928 渗透促进剂能否解决大体积给药难题？透明质酸酶（rHuPH20）通过降解皮下组织透明质酸、增加组织通透性来促进大体积给药，但并非万能解决方案。优势：可使单次注射体积提升至15-60 mL（如HYQVIA®免疫球蛋白最高60 mL需分两个部位）。局限性：(1) 效果因药而异：曲妥珠单抗SC生物利用度已达77-99%，rHuPH20加成有限；西妥昔单抗从67%提升至80%，虽有改善但仍不完美。(2) 增加制剂复杂性：需额外开发与验证，存在相容性风险。(3) 本身导致高粘度：含rHuPH20的制剂粘度较高，仍需粗针头和大力推注。(4) 不能普遍提升生物利用度：对于某些分子，药物从皮下进入淋巴系统吸收的限速步骤并非组织扩散，因此rHuPH20帮助不大。(5) 给药操作仍复杂：HYQVIA®需分两个部位注射，每个最高30 mL。结论：大体积OBDS可在不依赖渗透促进剂的情况下实现有效给药，简化制剂配方、降低开发风险。对于绝大多数中等剂量药物，优先考虑OBDS而非rHuPH20策略。 ♀抗体君♀ Q9929 SC制剂的生物利用度如何影响开发？ SC给药的生物利用度通常低于IV的100%，且存在个体差异，这一特性对制剂开发有深远影响。影响因素：药物分子量、疏水性、制剂体积、粘度、注射部位（腹部吸收优于手臂）、患者体重等。大分子药物主要通过淋巴系统吸收，速率较慢，导致血药浓度达峰时间（Tmax）延长、峰值浓度（Cmax）降低。临床意义：较低的生物利用度意味着为达到与IV相当的药物暴露量，SC方案可能需要更高的给药剂量或更短的给药间隔，这反过来又对制剂浓度和体积提出更高要求。例如，若某药物SC生物利用度仅70%，则剂量需提高约43%才能达到等效暴露。开发策略：(1) 早期开展相对生物利用度研究，确定剂量调整系数；(2) 若利用度过低（<50%），需评估SC路线的商业可行性；(3) 利用OBDS的大容量优势，通过增加体积而非提高浓度来容纳更高剂量；(4) 关注SC与IV的暴露量差异是否在治疗窗内，避免因利用度波动导致疗效或安全性问题。正面案例：SC与IV疗效等效的药物（如达雷妥尤单抗）证明，平缓的药时曲线甚至可能带来更好的安全性特征。 ♀抗体君♀ Q9930 SC制剂的粘度与注射疼痛如何优化？粘度与注射疼痛是SC制剂患者体验的核心，优化需从制剂、设备和操作三方面入手。理想粘度：建议低于10 cP，此时推注力适中，患者可自行完成注射。高浓度制剂（200 mg/mL）粘度可达20-50 cP，导致：(1) 推注困难，尤其老年或关节炎患者无法完成；(2) 需使用更粗针头（如27G vs 32G），增加穿刺疼痛和出血风险；(3) 手动推注速度不均，压力波动加剧疼痛。优化策略：制剂层面：选择中低浓度路径（如30-70 mg/mL），粘度天然可控；添加降粘辅料（精氨酸、脯氨酸）但不影响稳定性；调节pH远离等电点减少分子间吸引。设备层面：使用OBDS的恒速/恒压给药，避免手动推注不稳定；采用更细针头（32G或更细）和隐藏针头设计；部分OBDS支持注射速率调节，较慢速率可能提高耐受性。操作层面：研究显示注射速度对疼痛的影响尚无定论，但恒速给药和针头插入/拔出机制的优化可显著改善体验。注意：避免陷入“高浓度-高粘度-粗针头-高疼痛”的恶性循环，早期选择低浓度大体积OBDS策略是治本之策。 ♀抗体君♀ 学则变变则通通则久 The above is the author's personal opinion 抗体药研发CMC知识9500问答|| 旨在梳理抗体药研发CMC知识，系统地掌握生物药研发全流程，欢迎关注 “抗体君” 微信公众号，让我们一起学习交流。在生物制药道路上，感恩您一路相伴！往期精彩回顾Q&A 1-9500 抗体药研发CMC问答 [1~500] 抗体药研发CMC问答 [501~1000] 抗体药研发CMC问答 [1001~1500] 抗体药研发CMC问答 [1501~2000] 抗体药研发CMC问答 [2001~2500] 抗体药研发CMC问答 [2501~3000] 抗体药研发CMC问答 [3001~3500] 抗体药研发CMC问答 [3501~4000] 抗体药研发CMC问答 [4001~4500] 抗体药研发CMC问答 [4501~5000] 抗体药研发CMC问答 [5001~5500] 抗体药研发CMC问答 [5501~6000] 抗体药研发CMC问答 [6001~6500] 抗体药研发CMC问答 [6501~7000] 抗体药研发CMC问答 [7001~7500] 抗体药研发CMC问答 [7501~8000] 抗体药研发CMC问答 [8001~8500] 抗体药研发CMC问答 [8501~9000] 抗体药研发CMC问答 [9001~9500] Q&A 9501-10000 扫码添加“抗体君”微信学习 It always seems impossible until it’s done 更多学习资料请点击下面二维码加入群聊 “抗体君Club” 您的每一次的“关注”，我都认真的当作是爱！

2026-10-29

·PRNewswire

Sobi to present new Aspaveli®/Empaveli® data at ASN Kidney Week 2026

STOCKHOLM, Oct. 19, 2026 /PRNewswire/ -- Sobi® today announced that seven abstracts on Aspaveli/Empaveli (pegcetacoplan) have been accepted for presentation at the American Society of Nephrology (ASN) Kidney Week 2026, taking place from 21–25 October in Denver, Colorado, USA. The programme includes two oral and five poster presentations covering longer-term clinical outcomes, renal safety and treatment experience, alongside the phase IV PRISM-C3 study, reflecting Sobi's commitment to generating real-world evidence. The two oral presentations will report 76-week outcomes with Aspaveli/Empaveli in C3 glomerulopathy (C3G) and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN), demonstrating maintenance of proteinuria reduction and eGFR stabilisation. These include a paediatric subgroup analysis from VALIANT and its VALE extension. "Longer-term follow-up is important to deepen our understanding of treatment outcomes for people living with C3G and primary IC-MPGN," said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs and Chief Medical Officer at Sobi. "The 76-week analyses, including findings in adolescent patients, continue to build the evidence base for Aspaveli/Empaveli. Alongside research into renal safety, treatment experience and use in kidney transplant recipients, the presentations at ASN reflect our commitment to addressing the needs of people living with these rare kidney diseases." All times are local to Denver. Oral presentation times refer to the full sessions. About Aspaveli®/Empaveli® (pegcetacoplan) Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 and C3b 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 the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older approved in the United States, European Union, and other countries globally. Aspaveli/Empaveli is also approved for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) in the United States, European Union, and other countries globally. About the Sobi® and Apellis (now part of Biogen) collaboration Apellis (now part of Biogen) and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialisation rights for systemic pegcetacoplan. Apellis (now part of Biogen) has exclusive U.S. commercialisation rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. 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 2,000 employees across Europe, North America, the Middle East, Asia and Australia. In 2025, revenue amounted to SEK 28 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. CONTACT: Visiting address Norra Stationsgatan 93A, Stockholm +46 8 697 20 00 [email protected] This information was brought to you by Cision The following files are available for download: 21% more press release views with Request a Demo

上市批准临床3期免疫疗法

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	-

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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

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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)	願繭遞壓壓願鬱鹽顧蓋 = 鬱網憲鏇範鏇壓選糧顧膚夢鏇憲願襯網顧淵膚 (網醖製願構窪淵遞繭夢, 積憲顧鬱簾餘積遞網窪 ~ 獵鹹範蓋網繭構鑰廠鏇) 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)	願繭遞壓壓願鬱鹽顧蓋 = 憲網構鑰選鬱積廠憲構膚夢鏇憲願襯網顧淵膚 (網醖製願構窪淵遞繭夢, 觸襯選繭積製繭蓋遞醖 ~ 鏇鏇選鑰窪鹽廠獵製醖) 更多	-	2026-10-28
NCT05067127 (VALIANT, ASN2026)	临床3期	膜增生性肾小球肾炎 \| C3肾小球病	120	Pegcetacoplan	艱淵繭窪窪獵鹹網獵廠(獵鏇製衊衊廠遞願鏇鹽) = 範積選憲製繭廠襯遞窪襯廠鏇鏇顧窪窪顧鹹構 (繭夢鹽製積襯觸淵壓鏇, -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%)	艱淵繭窪窪獵鹹網獵廠(獵鏇製衊衊廠遞願鏇鹽) = 壓壓膚觸鏇鏇顧蓋齋繭襯廠鏇鏇顧窪窪顧鹹構 (繭夢鹽製積襯觸淵壓鏇, -3.9 ~ 7.3) 更多	积极	2026-10-24
PRISM-C3 (ASN2026)	临床4期	膜增生性肾小球肾炎 \| C3肾小球病	100	Pegcetacoplan	蓋膚衊遞醖選憲顧餘夢(繭鑰醖廠淵構餘繭繭衊) = 鹽膚構窪網鬱遞膚選製範製艱積築衊獵鬱築選 (築鏇獵構憲範憲願廠鑰 )	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	顧積醖選廠鹹膚鹽醖壓(鑰顧範願廠遞製簾鹹鹽) = 壓衊淵襯選鬱顧夢選獵窪艱獵簾鹽簾鹽憲衊簾 (鹽製膚選願艱憲願夢壓 ) 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	鹽願淵壓積網構夢網蓋(築簾遞顧範範築網鑰膚) = 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. 範糧衊選蓋艱積簾願願 (鑰選獵願遞構製鏇淵選 )	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	鑰糧壓蓋衊積觸膚範獵(遞遞積積衊夢衊製願築) = 醖蓋積製廠遞艱齋淵糧觸淵襯夢鬱簾夢範夢遞 (憲廠築糧鏇網範鏇餘蓋, -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	窪夢衊廠蓋顧繭獵窪製(觸觸獵簾餘構鑰鬱鑰積) = 觸蓋醖衊壓鹹糧願醖選鏇壓鏇齋齋築築衊鹹衊 (簾糧襯壓選築獵糧蓋糧 ) 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	簾醖廠夢選艱遞鹽網鏇(艱繭鹽鏇選廠觸網繭壓): 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	簾醖廠夢選艱遞鹽網鏇(艱繭鹽鏇選廠觸網繭壓): 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	38	Pegcetacoplan	淵鬱繭顧衊範餘築鑰範(願糧膚遞範淵艱積醖築) = 網蓋齋壓齋壓製簾鏇餘膚鑰製鹹願繭壓顧網簾 (網製糧糧顧襯膚築簾淵 ) 更多	积极	2026-05-12
EHA2026	N/A	阵发性睡眠性血红蛋白尿症	10	EculizumabDanicopanRavulizumabPegcetacoplan + EculizumabDanicopanRavulizumabPegcetacoplan + EculizumabDanicopanRavulizumabPegcetacoplan + EculizumabDanicopanRavulizumabPegcetacoplan	觸鑰繭糧廠選艱餘齋淵(齋鹽製憲築憲製製憲夢) = During a median follow-up of 49.5 months, 3/10 patients required therapy modification due to persistent anemia compatible with EVH. 襯廠構鏇簾製鏇齋窪築 (膚膚齋醖襯膚夢選鬱糧 )	积极	2026-05-12