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eculizumab (Ablyze)

✓ Approved

Cinnagen Co · C5 · 单克隆抗体

什么是 eculizumab？

eculizumab 是一种单克隆抗体，由Cinnagen Co研发。该药已获批，用于治疗相关适应症，给药途径：Injectable (Others)、Intravenous (IV)。

药物档案

商品名Ablyze
公司Cinnagen Co
药物类别单克隆抗体, 抗体
分子靶点C5
给药途径Injectable (Others), Intravenous (IV)
状态Approved
来源： ClinicalTrials.gov, Cinnagen Co

作用机制

分子靶点

eculizumab 作用于 1 个分子靶点：

C5complement C5 (C5b, C5D)
需要更深入的分析？Noah AI 可解释复杂机制并与同类药物比较。

治疗适应症

eculizumab 针对 4 个适应症，涉及 3 个治疗领域。

治疗领域疾病/病症分期
Blood and lymphatic system disordersHaemolytic uraemic syndrome✓ Approved
Nervous system disordersMyasthenia gravis✓ Approved
Renal and urinary disordersParoxysmal nocturnal haemoglobinuria✓ Approved
Nervous system disordersNeuromyelitis optica spectrum disorder✓ Approved

相关研究文献

PubMedEuropean journal of neurology2026-09-10

Practical Guidance on Initiating and Switching Targeted Immunotherapies in Generalised Myasthenia Gravis: A German-Austrian Expert Opinion Paper.

Meisel Andreas A, Marina Adela Della AD, Doksani Paolo P, Hagenacker Tim T et al.

The therapeutic landscape of generalised myasthenia gravis (gMG) has evolved substantially with the approval of targeted immunotherapies, including complement C5 inhibitors (C5-I) and neonatal Fc receptor inhibitors (FcRn-I). While pivotal trials have demonstrated marked efficacy in defined subgroups, real-world experience reveals more heterogeneous outcomes and raises questions about optimal patient selection, therapy sequencing and integration into clinical practice. In Germany and Austria, early access following regulatory approval has facilitated clinical experience over recent years. This expert opinion paper aims to combine current evidence with clinical experience to guide the use of C5-I and FcRn-I in everyday care. A panel of 18 neurologists from Germany and Austria, including two paediatric neurologists, evaluated study data and real-world experiences. Through structured discussion and a consensus process, they developed evidence- and experience-based recommendations on integrating targeted immunotherapies into existing treatment algorithms. The panel provides practical recommendations for managing (highly) active gMG in adults, focusing on C5-I (eculizumab, ravulizumab, zilucoplan) and FcRn-I (efgartigimod, rozanolixizumab). The statements cover initiation criteria, sequencing and switching within and between drug classes and transitions to intensified immunomodulatory therapies (rituximab, apheresis, intravenous or subcutaneous immunoglobulin), as well as special considerations for juvenile MG. This expert statement provides a practice-oriented framework integrating current evidence and clinical experience to support individualised therapeutic decision-making in gMG.

PMID 42717567
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PubMedNephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association2026-09-09

Efficacy and safety of eculizumab-combined therapy for patients with anti-GBM disease.

Lang Xiabing X, Hong Xizhen X, Wu Weifei W, Huang Xiaohan X et al.

The study aimed to evaluate the efficacy and safety of eculizumab in addition to conventional therapy for anti-glomerular basement membrane (anti-GBM) disease presenting rapidly progressive glomerulonephritis (RPGN). 42 patients with anti-GBM disease presenting with RPGN were included in this multicenter retrospective cohort study. All patients received intensive immunosuppressive therapy including intravenous methylprednisolone pulses followed by maintenance prednisone, in combination with at least two of the following: intravenous cyclophosphamide, therapeutic plasma apheresis, rituximab, or eculizumab. The cohort was stratified into two groups: the eculizumab group (n = 13) which received eculizumab (900 mg once weekly for 1-5 doses) in combination with other therapies, and the control group (n = 29) which did not receive eculizumab. Kidney survival at 12 weeks and 24 weeks, and adverse events (infections, metabolic alterations, laboratory abnormalities), ascertained via clinical and laboratory data. At 12 weeks, the eculizumab group showed significantly higher kidney survival compared with the control group (69.2% vs. 31.0%, p = 0.021). At 24 weeks, kidney survival remained numerically higher in the eculizumab group (66.7% vs. 40.7%, p = 0.081). Kaplan-Meier curves for renal survival by the Gehan-Breslow-Wilcoxon test, which places greater emphasis on early events, revealed a statistically significant difference between the groups (χ² = 4.137; p = 0.042); however, the difference assessed by the log-rank test did not reach statistical significance (χ² = 3.491; p = 0.062). There were no significant differences in the overall incidence of infections or other adverse events. Combined therapy with eculizumab showed improved early kidney survival for anti-GBM nephritis at 12 weeks, and did not increase the risk of adverse events. Prospective, large-sample studies with long-term follow-up are needed to validate its efficacy and safety.

PMID 42714831
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PubMedTherapeutic advances in hematology2026-09-08

A real-world, multi-center, prospective, observational study for paroxysmal nocturnal haemoglobinuria (PNH) in China: Baseline characteristics, disease burden and treatment patterns.

Han Bing B, Liu Hui H, He Guangsheng G, Jia Jinsong J et al.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare life-threatening hematologic disorder with high thromboembolic mortality. In the context of large patient population and limited use of complement inhibitors, the clinical characteristics and disease progression of patients in China have not been well studied. The China PNH Registry was initiated to advance understanding of the disease by collecting data and describing PNH disease burden, progression, and clinical outcomes with different medical interventions. This is a multi-center, prospective observational study in patients with PNH regardless of treatment for PNH. This real-world study enrolled 716 PNH patients irrespective of treatment, collecting baseline demographics, clinical/laboratory data, and treatment patterns (including eculizumab dosing and safety) for descriptive analyses across PNH subtypes. 52.0% of the enrolled patients had classic PNH, 47.2% had bone marrow failure (BMF/PNH), and 0.8% had subclinical PNH. Classic PNH patients had a higher proportion of PNH red blood cells (RBCs) (39.0% vs. 18.0%), PNH neutrophils (86.1% vs. 52.4%), and PNH monocytes (90.2% vs. 47.7%) than BMF/PNH patients. 343/496 patients had LDH > 1.5 ULN, with a higher proportion in classic PNH than BMF/PNH (74.7% vs. 63.1%). 582 patients had at least one of the PNH-related symptoms, the most common being fatigue (63.7%), red/dark urine (46.6%). Although eculizumab has become a reimbursable complement inhibitor in China, only 28.5% of the patients received eculizumab treatment during the study period. The other main treatment methods were supportive care (46.6%), corticosteroids (28.1%), and RBC transfusion (7.5%). Current data revealed a gap between real-world practice and guideline recommendations, indicating that standard treatment - particularly complement inhibitors - remains underutilized despite being essential for improving long-term patient outcomes. The China Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry. NCT06154512.

PMID 42707331
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PubMedPediatric transplantation2026-09-08

An Approach to Antibody-Mediated Rejection in Pediatric Liver Transplantation.

Jaramillo Catalina C, Randhawa Parmjeet P, Narang Amrita A, Gareau Alison J AJ et al.

Antibody-mediated rejection (AMR) is an important but infrequent cause of pediatric liver allograft injury. There is a lack of standardized guidance, and treatment approaches vary across institutions. A writing group from the Society of Pediatric Liver Transplantation conducted a comprehensive review of the published literature and collected institutional guidance documents from pediatric transplant centers across the United States to synthesize current diagnostic and management approaches to AMR in pediatric liver transplant recipients. AMR is diagnosed by the presence of characteristic histologic injury, C4d deposition, circulating donor-specific antibody (DSA), and exclusion of alternative causes of graft dysfunction, including T cell-mediated rejection. Management strategies focus on reducing circulating DSA through therapeutic plasma exchange and suppressing ongoing immune activation with corticosteroids and intravenous immunoglobulin. In severe or refractory cases, targeted therapies such as rituximab, bortezomib, and eculizumab may be considered. A stepwise diagnostic and management algorithm is presented. This resource outlines a practical approach to the diagnosis and management of AMR in pediatric liver transplantation, serving as a reference for busy clinicians who may encounter this serious but underrecognized cause of graft dysfunction and loss.

PMID 42708530
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PubMedClinical kidney journal2026-09-08

Current practices in antibiotic prophylaxis for patients on complement inhibitors: a multicenter survey across ERN ERKnet centers.

Sciascia Savino S, Terzolo Edoardo E, Fenoglio Roberta R, Roccatello Dario D et al.

Complement inhibitors such as eculizumab and ravulizumab are increasingly used to treat rare complement-mediated diseases. While effective, these therapies impair host defenses against encapsulated bacteria, leading to a heightened risk of invasive infections. Although vaccination is standard, the role and implementation of antibiotic prophylaxis remain inconsistent across clinical settings. A survey was conducted among European Reference Network for Rare Kidney Diseases (ERN ERKNet) centers to assess current practices regarding antibiotic prophylaxis in patients receiving complement-inhibition therapy. The survey addressed four clinical scenarios-pediatric vs. adult, transplant recipient vs. non-transplant-and explored three domains: indications for prophylaxis, choice of antibiotic, and duration. Twenty-seven centers from 16 European countries participated. Systematic prophylaxis was widely endorsed, particularly for pediatric patients and transplant recipients. Penicillin-class antibiotics were preferred as first-line agents across all groups. In pediatric settings, phenoxymethylpenicillin and amoxicillin were most commonly used, whereas adult regimens included penicillin or fluoroquinolones for those with allergies. Most respondents recommended continuing prophylaxis throughout the duration of complement-inhibition therapy, regardless of vaccination status. The survey revealed areas of heterogeneity in practice, especially regarding allergy management and the duration of post-vaccination coverage. This ERKNet survey identifies prevailing practices and variation in antibiotic prophylaxis for patients receiving complement-inhibition therapy. Based on these findings, we propose consensus guidance to support harmonized, risk-adapted prophylaxis strategies across pediatric and adult populations. These guidance statements aim to address an important unmet need in infection prevention among complement-inhibited patients.

PMID 42707644
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PubMedMultiple sclerosis and related disorders2026-09-04

A systematic review and meta-analysis of the efficacy and safety of eculizumab in AQP4-IgG-positive neuromyelitis optica spectrum disorder.

Liu Wanfen W, Wu Jiaoyang J, Liu Zhuyun Z, Lu Hongji H et al.

Neuromyelitis optica spectrum disorder (NMOSD) is an extremely uncommon autoimmune inflammatory condition affecting the central nervous system with clinical features including recurrent episodes of optic neuritis and transverse myelitis. Complement-dependent astrocyte damage is the predominant mechanism involved in the development of NMOSD, which is positive for anti-aquaporin-4 antibodies (AQP4). Eculizumab, an inhibitor of the complement C5 protein, shows efficacy in preventing relapses. Objective Evaluating the effectiveness and safety of eculizumab in AQP4-IgG seropositive NMOSD subjects, specifically regarding preventing relapse in addition to annualized relapse rate (ARR), neurological disability, and SAEs METHODS: The systematic review and meta-analysis were done according to PRISMA guidelines. A search of PubMed, Scopus, Web of Science, Embase, and Cochrane Library was performed from inception until March 2026 for RCTs investigating eculizumab in AQP4-IgG-seropositive NMOSD patients. The time to first adjudicated relapse and ARR were chosen as primary outcomes, while the EDSS scores and SAEs were selected as secondary outcomes. Phase III RCTs were included in the study RESULTS: Eculizumab significantly reduced the risk of adjudicated relapse compared with placebo or conventional immunosuppressive therapy (HR = 0.07; 95% CI: 0.03-0.18; p < 0.001). It also significantly reduced ARR (RR = 0.15; 95% CI: 0.08-0.29; p < 0.001). More than 95% of patients receiving eculizumab remained relapse-free during follow-up. EDSS scores generally remained stable, suggesting preservation of neurological function. The available evidence indicated an acceptable safety profile, although the limited number of trials and relatively short follow-up periods restrict conclusions regarding long-term safety CONCLUSION: Eculizumab decreases relapse rates and ARR in AQP4-IgG-positive NMOSD patients and is considered safe. Nevertheless, the lack of RCTs, small sample sizes, heterogeneity in follow-up periods, and limited data on long-term safety and cost-effectiveness require additional studies.

PMID 42697106
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