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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

PubMedNeurology and therapy2026-10-24

Current and Emerging Therapies for Generalized Myasthenia Gravis: Toward Precision Medicine.

Yoshikawa Masaaki M, Ide Toshihiro T, Koike Haruki H

The therapeutic landscape of myasthenia gravis (MG) has changed substantially with advances in immunopathogenesis and the introduction of targeted, mechanism-based immunotherapies. For decades, treatment relied primarily on corticosteroids and conventional immunosuppressive agents, which are effective but often limited by delayed onset, long-term toxicity, and incomplete disease control. This review summarizes current and emerging therapies for generalized MG, focusing on mechanisms of action, clinical efficacy, safety, and practical considerations, while also addressing real-world evidence, treatment switching and sequencing, and factors relevant to individualized decision-making. Complement-targeted therapies, including eculizumab, ravulizumab, zilucoplan, gefurulimab, and the emerging RNA interference-based agent cemdisiran, and neonatal Fc receptor (FcRn) inhibitors, including efgartigimod, rozanolixizumab, and nipocalimab, have substantially expanded therapeutic options. B cell-targeted therapies, particularly rituximab and inebilizumab, have also broadened the therapeutic landscape, especially for refractory disease and muscle-specific kinase antibody-positive MG. Emerging approaches, including chimeric antigen receptor T cell and chimeric autoantibody receptor T cell therapies, plasma cell-directed therapies, regulatory T cell-based therapies, and antigen-specific immune tolerance strategies, may further improve long-term disease control by targeting the underlying autoimmune process. Important unmet needs remain, including limited treatment accessibility, insufficient evidence for specific patient populations, and the lack of validated predictive biomarkers. In the absence of direct head-to-head trials or validated predictive biomarkers, individualized treatment selection should integrate autoantibody status, disease activity and urgency, clinical characteristics, prior treatment response and toxicity, comorbidities and safety considerations, treatment burden, reproductive considerations, patient preferences, and access. Future progress will depend on the development and validation of predictive biomarkers to enable biomarker-guided precision medicine, together with advances in targeted immunotherapy and immune tolerance-inducing strategies, with the ultimate goal of achieving durable remission and improving long-term outcomes and quality of life.

PMID 42920067
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PubMedFrontiers in immunology2026-10-23

Clinical study of eculizumab with or with not tacrolimus in the treatment of generalized myasthenia gravis.

Yang Bin B, Wang Jing J, Zhao Xiuli X, Jing Chuya C et al.

Eculizumab, an antibody that inhibits the complement system, and the immunosuppressant Tacrolimus are both authorized for treating patients with generalized myasthenia gravis (GMG). This study seeks to evaluate and compare the clinical effectiveness of Eculizumab with or with not Tacrolimus in the treatment of generalized myasthenia gravis. A retrospective study was performed involving 95 patients with GMG treated with Eculizumab at three hospitals in China. Among them, 80 patients were included in the analysis: 57 received Eculizumab alone, while 23 were treated with a combination of Eculizumab and Tacrolimus. The main outcome assessed was the percentage of GMG patients who reached Minimal Symptom Expression (MSE). Secondary outcomes included changes in MG-Activities of Daily Living (MG-ADL) scale, Quantitative Myasthenia Gravis (QMG) score, CH50 concentration, and doses of pyridostigmine and prednisolone. Additionally, the safety profiles of both treatment groups were examined. In the main outcome, a higher proportion of patients reached MSE status when treated with a combination of Eculizumab and Tacrolimus compared to Eculizumab alone (Eculizumab: 29 patients, 50.9% vs Eculizumab and Tacrolimus: 18 patients, 78.3%; p<0.05). Regarding secondary outcomes, both treatment groups showed significant improvements in clinical symptoms, as indicated by notable reductions in QMG and MG-ADL scores, along with a significant reduction in prednisolone dosage. Furthermore, analysis of secondary outcome parameters revealed no significant differences between the two groups (p>0.05). The decrease in complement CH50 concentration is accompanied by a decrease in QMG and MG-ADL scores, indicating that Eculizumab treats GMG through the mechanism of complement system. In terms of security, hyperuricemia was more commonly associated with the combination therapy of Eculizumab and Tacrolimus than with Eculizumab alone. Our research indicates that Eculizumab can effectively reduce disability in GMG patients by inhibiting complement system activation under real-world clinical conditions in China. We observed a correlative link between Eculizumab and Tacrolimus combination treatment and an increased probability of achieving MSE status, in comparison with Eculizumab monotherapy. However, combination therapy is associated with a higher risk of elevated serum uric acid among GMG patients, which necessitates regular close monitoring of renal function.

PMID 42908923
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PubMedZeitschrift fur Rheumatologie2026-10-23

[Gastrointestinal manifestations in idiopathic inflammatory myopathies : Dysphagia, intestinal vasculopathy and pneumatosis cystoides intestinalis].

Seitz Pascal P

In idiopathic inflammatory myopathies (IIM), gastrointestinal manifestations are rare and poorly recognized apart from the relatively common dysphagia. Oropharyngeal or esophageal dysphagia in IIM is associated with aspiration and malnutrition and responds well to conventional immunomodulation except for inclusion body myositis. The rare intestinal vasculopathy in nuclear matrix protein 2 (NXP2)-associated dermatomyositis affects both children and adults, causing life-threatening perforation and bleeding from the esophagus to the colon with a high mortality. It responds poorly to standard immunomodulation. Experimental treatment approaches with Janus kinase (JAK) inhibitors (by interferon inhibition), vedolizumab, and the C5 inhibitor eculizumab have recently shown promising results. Pneumatosis cystoides intestinalis, also a rarity, manifests as gas-filled submucosal cysts primarily in the small and large bowels and is often but not always an asymptomatic incidental finding in IIM amenable to conservative management. The IIM can involve dysmotility and hypomotility throughout the gastrointestinal tract, potentially leading to intestinal pseudo-obstruction and bacterial overgrowth with malnutrition. Notably, elevated aspartate aminotransferase (AST) and alanine transaminase (ALT) in IIM with high creatine kinase typically reflect skeletal muscle release rather than the manifestation of a hepatopathy.

PMID 42918901
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PubMedThe journal of applied laboratory medicine2026-10-23

Laboratory Diagnosis of Complement-Mediated Diseases: A Practical Overview.

Valentini Alessandra A, Bernardini Sergio S

Complement is not a niche test area. An expanding group of rare diseases, including hereditary angioedema, atypical hemolytic uremic syndrome, C3 glomerulopathy, and paroxysmal nocturnal hemoglobinuria, now has approved targeted therapies, and the laboratory is central to both diagnosis and treatment monitoring. Yet complement testing remains poorly standardized across institutions, and its preanalytical fragility makes it uniquely susceptible to errors that other immune assays tolerate. This review offers a working framework for complement diagnostics in the clinical laboratory. We describe the 3 activation pathways and their key regulators and then outline a tiered diagnostic panel: functional assays of classical pathway activity (CH50), alternative pathway activity, protein quantitation (C3, C4, C1q, complement factor H, complement factor I, and C1 inhibitor antigen and function), and activation products (C3d, C4d, Bb, and sC5b-9), and disease-specific autoantibodies. Diagnostic algorithms are presented for the main complement-mediated conditions, with emphasis on the serological features that distinguish 1 disease from another. Preanalytical requirements receive dedicated attention as tube selection, centrifugation temperature, processing delay, and freeze conditions all materially affect results. A practical approach to laboratory-developed test validation is outlined, and we address the interpretive challenges introduced by approved complement inhibitors (eculizumab, ravulizumab, pozelimab, zilucoplan, danicopan, iptacopan, pegcetacoplan, and sutimlimab), whose effects on routine complement assays are not always straightforward. Future directions, including multiplex platforms and artificial intelligence-assisted interpretation, are briefly considered. A well-structured complement laboratory, based on rigorous preanalytical practices and validated assays, provides diagnostic information not available from other tests. This review could serve as a practical reference for laboratory clinical biochemists building or optimizing complement diagnostics in their setting.

PMID 42910908
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PubMedJournal of clinical medicine2026-10-16

Legionella Septic Cardiomyopathy Requiring Venoarterial Extracorporeal Membrane Oxygenation Followed by Presumed Complement-Mediated Thrombotic Microangiopathy: A Case Report.

Liu Hui H, Zhang Chengduo C, Peng Shengjia S, Li Dan D et al.

Background:Legionella pneumophila rarely causes profound myocardial dysfunction and malignant ventricular arrhythmias. Persistent thrombocytopenia and acute kidney injury during recovery may additionally raise concern for thrombotic microangiopathy (TMA), although distinguishing complement-mediated disease from secondary causes in critically ill patients is challenging. Case Presentation: A 64-year-old man presented after five days of fever with recurrent ventricular tachycardia/ventricular fibrillation, cardiac arrest, and refractory shock. Echocardiography demonstrated diffuse biventricular hypokinesia with a left ventricular ejection fraction (LVEF) of 18%, while urgent coronary angiography showed no acute coronary occlusion. Venoarterial extracorporeal membrane oxygenation (VA-ECMO), intra-aortic balloon counterpulsation, and organ support were initiated. Levofloxacin was administered from admission, before sputum sequencing identified L. pneumophila. VA-ECMO was removed after four days. Persistent anaemia and thrombocytopenia with progressive acute kidney injury despite clinical improvement prompted evaluation for thrombotic microangiopathy. ADAMTS13 activity was 92%, soluble C5b-9 was elevated to 648 ng/mL, and schistocytes were not identified. Presumed complement-mediated TMA was treated with four weekly doses of eculizumab, followed by haematological and renal improvement. Sepsis, extracorporeal support, transfusions, and gastrointestinal bleeding confounded diagnosis and treatment attribution. At three months, LVEF was 59%, platelet count 237 × 109/L, and creatinine 97 µmol/L. Conclusions: VA-ECMO may bridge selected patients with severe septic myocardial dysfunction to recovery. Persistent cytopenias and kidney injury warrant structured TMA evaluation. In critically ill patients, complement-mediated TMA requires integrated assessment rather than reliance on a single complement biomarker or clinical response to complement inhibition.

PMID 42881879
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PubMedJournal of clinical medicine2026-10-16

Congenital/Inherited CD59 Deficiency: A Systematic Review.

Alwahiby Lama L, Alsuhim Lanah L, Alzahrani Manal M, Albaloshi Jalnar J et al.

Background: Inherited CD59 deficiency is a rare autosomal recessive complement-mediated disorder caused by biallelic loss-of-function variants in the CD59 gene. It typically presents in infancy with Coombs-negative hemolytic anemia, relapsing demyelinating polyneuropathy, and recurrent ischemic strokes. Diagnosis is often delayed due to overlapping with more common neurological and hematological disorders. The present study was carried out to systematically review the literature on congenital CD59 deficiency, focusing on genotype-phenotype correlations, geographic distribution, clinical presentation, diagnostic approaches, and treatment outcomes. Methods: Eligible studies included case reports and case series of genetically or functionally confirmed inherited CD59 deficiency. Demographic, molecular, clinical, and therapeutic data were extracted and analyzed descriptively. Results: 20 reports included. Evidence remains mainly limited to case reports and small case series. The disorder demonstrates a severe and heterogeneous phenotype with predominant neuromuscular involvement, frequently associated with hematologic and cerebrovascular manifestations. Most reported cases originate from regions with high consanguinity, particularly the Middle East and Asia, suggesting possible founder effects and underrecognized prevalence. Exploratory analysis suggested a possible relationship between predicted variant functional impact and clinical severity. However, given the small and heterogeneous evidence base, this finding should be considered hypothesis-generating. Responses to conventional immunomodulatory therapies are variable and often transient. Eculizumab has shown clinical benefit in some patients, although treatment responses remain inconsistent. Conclusions: Congenital CD59 deficiency is a severe and underrecognized complement-mediated disorder with major diagnostic and therapeutic challenges. Current evidence remains insufficient to establish definitive genotype-phenotype correlations or standardized management strategies. Increased clinical awareness, early integration of functional and genetic testing, and further studies are needed to improve diagnosis and optimize treatment outcomes.

PMID 42882210
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