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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

PubMedCureus2026-07-31

Severe Class III Lupus Nephritis With Concurrent Thrombotic Microangiopathy and Suspected Atypical Hemolytic Uremic Syndrome Requiring Complement Blockade: A Complex Multisystem Presentation.

Pokharel Nishma N, Alvi Arsalan A, Best Alejandro A

Systemic lupus erythematosus (SLE) is frequently complicated by lupus nephritis (LN), but the coexistence of thrombotic microangiopathy (TMA) represents a rare and severe manifestation associated with poor renal outcomes. Differentiating between SLE-associated TMA and primary complement-mediated atypical hemolytic uremic syndrome is diagnostically challenging but critical, as it dictates the use of targeted therapies such as terminal complement inhibitors. A 36-year-old female with SLE, lost to follow-up for two years, presented with fatigue, seizures, and anuric renal failure. Laboratory studies revealed severe bicytopenia, metabolic acidosis, and hemolysis with normal ADAMTS13 activity. A renal biopsy confirmed International Society of Nephrology/Renal Pathology Society Class III LN with prominent superimposed TMA. Despite intensive management with high-dose steroids, plasma exchange, and hemodialysis, the patient's refractory state necessitated the initiation of eculizumab. The clinical course was further complicated by a positive direct anti-globulin test (C3 positive/IgG negative), requiring intravenous immunoglobulin for a suspected secondary immune-mediated anemia. This case illustrates the diagnostic complexity of LN-TMA and the therapeutic necessity of a multidisciplinary approach. It underscores the importance of early renal biopsy and the timely utilization of complement inhibition in refractory cases to mitigate irreversible renal damage.

PMID 42534175
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PubMedClinical pharmacology and therapeutics2026-07-31

Pediatric Extrapolation in Neuropsychiatric Drug Development: Leveraging Pharmacokinetic and Pharmacodynamic Strategies to Facilitate FDA Approvals.

Nookala Anantha Ram AR, Murphy William A WA, Panse Nimishraj N, Bewernitz Michael M et al.

Pediatric extrapolation enables the application of adult efficacy data to pediatric populations when sufficient similarity is established in disease pathophysiology, pharmacologic response, and drug exposure. When these conditions are met, extrapolation can reduce or eliminate the need for dedicated pediatric efficacy trials, facilitating more efficient pediatric drug development. In this paper, we examined US Food and Drug Administration (FDA) pediatric drug approvals in neurology, analgesia, and psychiatry from 2014 to 2025 that utilized exposure matching to support extrapolation approaches. We identified 33 unique drug products that successfully implemented pharmacokinetic-based exposure matching strategies across neuropsychiatric indications. Population pharmacokinetic modeling emerged as the predominant strategy for pediatric dose optimization, incorporating developmental considerations such as allometric scaling and enzyme maturation functions. FDA General Advice Letters proved instrumental in facilitating adoption of complete pediatric extrapolation approaches, demonstrating the critical role of regulatory guidance in expediting pediatric drug development. Case examples spanned broad age ranges, from mechanistically informed neonatal applications utilizing enzyme maturation modeling (risdiplam) to adolescent populations where adult dosing regimens proved adequate (brexanolone, nipocalimab). Additionally, pharmacodynamic biomarkers were pivotal in pediatric extrapolation of eculizumab and nipocalimab efficacy for the treatment of generalized myasthenia gravis, highlighting promising future directions for the field. These cases demonstrate how achieving comparable pharmacokinetics and, when necessary, pharmacodynamics profiles between adult and pediatric populations can support efficient regulatory approval without dedicated pediatric efficacy studies. The regulatory pathways established for select neuropsychiatric indications provide a compelling framework for expanding extrapolation approaches to other therapeutic areas.

PMID 42533553
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PubMedAutoimmunity reviews2026-07-31

Aberrant neutrophil and complement activation in thrombotic microangiopathies in pregnancy - Is there a missing link?

Zaimi Maria M, Kambas Konstantinos K, Marinaki Smaragdi S, Theodora Marianna M et al.

Pregnancy-related acute kidney injury is a major global health burden associated with increased maternal and fetal morbidity and mortality. Thrombotic microangiopathies (TMA) occurring in pregnancy or postpartum include severe preeclampsia/Hemolysis Elevated Liver Enzymes Low Platelets (HELLP) syndrome, atypical Hemolytic Uremic Syndrome (aHUS) and Thrombotic Thrombocytopenic Purpura (TTP). Severe acute kidney injury and progression to chronic kidney disease are particularly associated with complement-mediated thrombotic microangiopathy/atypical hemolytic uremic syndrome and may also complicate severe preeclampsia/HELLP syndrome, while renal involvement is usually less prominent in TTP. Interestingly, preeclampsia, HELLP syndrome, aHUS, TTP, lupus nephritis and antiphospholipid syndrome share common autoimmune pathogenic mechanisms, as both are characterized by exaggerated neutrophil activation and increased neutrophil extracellular traps (NETs) release, as well as complement activation. In an era of targeted therapies, the lack of treatment of preeclampsia and HELLP results from the limited understanding of the underlying pathogenetic mechanisms. Eculizumab, a C5 inhibitor that targets the complement, significantly reduced the risk of ESKD in women with pregnancy-associated aHUS, supporting the therapeutic relevance of complement modulation. In this review, we performed a narrative synthesis of established and emerging experimental, translational and clinical data on pregnancy-associated TMAs, summarizing current evidence on the interaction between neutrophil dysregulation, NET formation, complement activation and endothelial injury in pregnancy-associated thrombotic microangiopathies. A better understanding of the neutrophil-NET-complement axis may help refine diagnostic distinctions among overlapping pregnancy-associated thrombotic microangiopathies and facilitate the development of more targeted therapeutic strategies.

PMID 42532252
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PubMedKidney medicine2026-07-30

Renal-Limited Thrombotic Microangiopathy in Infants: A Case Series.

Wang Haiyan H, Luo Shuting S, Wang Sijin S, Zhang Nisi N et al.

Thrombotic microangiopathy (TMA), an intractable disease, can present in a renal-limited mode. Data on infantile-onset renal-limited TMA (RL-TMA) is lacking. This study describes 5 cases of RL-TMA in infants. Case series. Five patients with infantile-onset RL-TMA who were admitted and/or followed up at Nanfang Hospital, Southern Medical University, from January 2024 to June 2025. Clinical data of 5 patients were collected. Amino acid conservation was analyzed by Clustal Omega, the secondary structure of the mutants was predicted by PolyPhen-2 and PHYRE2, and tertiary structure was predicted by AlphaFold3 and ChimeraX. All patients presented with acute nephritic syndrome with hematuria and proteinuria with onset in infancy, but without hemolytic anemia or thrombocytopenia. Corticosteroid treatment was attempted in 3 patients with no response. Renal pathology revealed microthrombi; segmental endothelial cells that swelled with widening of the subendothelial gap and/or mesangiolysis were observed. C3 heterozygous missense variant (c.2184 C>T, p.Cys728X), CD46 homozygous variant c.614A>G (p.Glu205Gly), and CFI heterozygous variant c.610A>G (p.Met204Val) were detected in 3 patients, respectively. Possible activation of alternative complement pathway (AP) was indicated in all 4 tested patients. Complete remission was achieved in 2 patients, and partial response was observed in another 2 patients who received eculizumab therapy. The patient who harbored the CFI variation and did not receive eculizumab therapy progressed to kidney failure at 11 months of age. Functional verification of genetic variants of C3, CD46 and CFI in this study were not implemented. AP activation-related factors were not checked in patient 5. RL-TMA can occur in infancy, which has not been reported previously. Activation of the AP may be the common cause of infantile-onset RL-TMA. Its diagnosis and therapy are challenging. Promising outcomes can be achieved with eculizumab therapy.

PMID 42529196
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PubMedJournal of critical care2026-07-30

Terminal complement inhibition is associated with renal, hematologic, and thrombotic complications during septic shock: A global propensity score-matched cohort study.

Vahldieck Carl C, Radermacher Philipp Bernd PB, Steinmeier Philip P, Fujita Buntaro B et al.

Terminal complement inhibition with eculizumab or ravulizumab alters host defense and vascular-immune interactions. However, its impact on outcomes during septic shock remains unclear. We investigated the association between terminal complement inhibition and clinical outcomes in patients with septic shock. We conducted a retrospective propensity score-matched cohort study using the TriNetX Global Collaborative Network, a large federated network of electronic health record data from healthcare organizations worldwide. Adult patients with septic shock and an underlying indication for C5 inhibition (atypical hemolytic uremic syndrome, myasthenia gravis, neuromyelitis optica spectrum disorder, or paroxysmal nocturnal hemoglobinuria) were stratified by treatment with eculizumab or ravulizumab. Propensity score matching balanced demographics, comorbidities, underlying disease indications, and severity proxies, yielding two cohorts of 638 patients each. Clinical outcomes were assessed over 31 days, with an additional 7-day sensitivity analysis. Patients receiving complement inhibition had higher risks of thrombocytopenia (20.3% vs 8.9%, p < 0.001), acute kidney injury (25.0% vs 13.3%, p < 0.001), and thrombotic disorders (7.8% vs 4.7%, p = 0.021). Renal replacement therapy (8.3% vs 5.1%, p = 0.026) and major adverse cardiovascular events (13.7% vs 9.2%, p = 0.011) were also more frequent. Time-to-event analyses confirmed these findings, and the overall outcome pattern remained consistent in the 7-day sensitivity analysis. Thirty-day mortality did not differ between the matched cohorts. In septic shock, terminal complement inhibition was associated with increased renal, hematologic, and thrombotic complications without a significant difference in short-term mortality. These findings identify a clinically vulnerable, currently uncommon but increasingly relevant patient population.

PMID 42526359
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PubMedCureus2026-07-30

Atypical Hemolytic Uremic Syndrome With Hypocellular Bone Marrow: A Report of a Rare Case.

Soren Champai R CR, Vasudevan Vibin K VK, Sharma Rachna R, Lal Naresh N

Atypical hemolytic uremic syndrome (aHUS) is not a common thrombotic microangiopathy (TMA) characterized by microangiopathic hemolytic anemia, thrombocytopenia, and renal impairment, often due to dysregulation of the alternative complement pathway. Bone marrow study in HUS is classically hypercellular. However, hypo-cellularity is rarely encountered. We report a case of a five-year-old boy who presented with severe pallor, thrombocytopenia, hypertension, and signs of TMA, including schistocytosis and renal impairment. Infectious and autoimmune causes were excluded. Initial management with plasma exchange and other supportive measures showed no improvement. ADAMTS13 activity was within normal limits, which excluded thrombotic thrombocytopenic purpura (TTP). Further investigations, including next-generation sequencing and anti-complement factor H (anti-CFH) antibody tests, were negative. To our surprise, bone marrow biopsy revealed markedly hypocellular marrow (10-15% cellularity), indicative of aplastic anemia. Despite escalation of therapy with steroids, intravenous immunoglobulin (IVIG), and eculizumab, the child showed no response and succumbed to a pulmonary hemorrhage. The case highlights a rare and fatal presentation of aHUS associated with bone marrow hypoplasia, an uncommon and diagnostically challenging phenotype. It underscores the need for high clinical suspicion, early bone marrow evaluation in refractory cases, and the importance of timely access to complement-targeted therapies. The coexistence of aHUS and aplastic anemia suggests a potential novel pathophysiologic overlap warranting further investigation.

PMID 42529101
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