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EC

eculizumab (Ablyze)

✓ Approved

Cinnagen Co · C5 · Monoclonal Antibodies

What is eculizumab?

eculizumab is a monoclonal antibodies developed by Cinnagen Co. It is approved for therapeutic indications via injectable (others) or intravenous (iv).

Drug Profile

Brand NamesAblyze
CompanyCinnagen Co
Drug ClassMonoclonal Antibodies, Antibody
Molecular TargetC5
RouteInjectable (Others), Intravenous (IV)
StatusApproved
Sources: ClinicalTrials.gov, Cinnagen Co

Mechanism of Action

Molecular Targets

eculizumab acts on 1 molecular target:

C5complement C5 (C5b, C5D)
Want deeper analysis?Noah AI can explain complex mechanisms and compare to similar drugs.

Therapeutic Indications

eculizumab is developed for 4 unique indications across 3 therapeutic areas.

Therapeutic AreaConditionPhase
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

Related Research Articles

PubMedHematology reports2026-07-24

Clinical Spectrum and Differential Diagnosis of Adult Thrombotic Microangiopathies: Real-World Experience from a Tertiary Referral Center.

Kırkayak Nazlı Pelin NP, Ozet Gulsum G, Dagdas Simten S, Ceran Funda F et al.

Background/Objectives: Thrombotic microangiopathies are rare, life-threatening hematological disorders characterized by microangiopathic hemolytic anemia, thrombocytopenia, and end-organ injury. This study was conducted in a setting where ADAMTS13 activity testing became available only from 2015 onward and complement-targeted therapy (eculizumab) had limited accessibility throughout most of the study period, conditions that shaped both diagnostic classification and treatment outcomes. Their clinical presentation, treatment response, and prognosis vary according to etiology, making early recognition and subtype classification clinically important. This study aimed to evaluate the etiological distribution, clinical features, treatment responses, and outcomes of adult patients with thrombotic microangiopathy at a tertiary-center real-world cohort. Methods: This retrospective cohort study included 47 adult patients (≥18 years) hospitalized with thrombocytopenia and microangiopathic hemolytic anemia (MAHA) in a nine-year period. Patients were classified as thrombotic thrombocytopenic purpura (TTP), hemolytic uremic syndrome (HUS), or secondary TMA based on clinical and laboratory evaluation. Demographic characteristics, clinical manifestations, laboratory parameters, treatments, and outcomes were analyzed. Results: The mean age was 45.3 ± 15.2 years, and 72.3% of patients were female. Primary thrombotic microangiopathy accounted for 74.5% of cases, including thrombotic thrombocytopenic purpura in 53.2% and hemolytic uremic syndrome in 21.2%; secondary thrombotic microangiopathy accounted for 25.5%. Hemodialysis was required in all patients with hemolytic uremic syndrome compared with 16% of those with thrombotic thrombocytopenic purpura. The complete response rate was 74.5%, and in-hospital mortality was 25.5%. In multivariable Cox regression analysis, treatment non-response and reduced post-treatment estimated glomerular filtration rate independently predicted mortality. Conclusions: Adult TMAs are characterized by considerable etiological and clinical heterogeneity, which makes differential diagnosis challenging, particularly in settings where access to contemporary diagnostic tests and targeted treatments is limited. In this cohort, in the absence of ADAMTS13 testing, TTP was the most frequent subtype, while treatment non-response and renal impairment emerged as the main factors associated with mortality. These findings emphasize the need for early clinical recognition and careful subtype-based differential diagnosis, which will reduce morbidity and mortality by permitting rapid initiation of pathophysiology-based appropriate interventions, i.e., PEx, immune suppression and caplacizumab for immune TTP and anti-complement therapy for aHUS, and limiting the inappropriate use of PEx with its complications, including sepsis.

PMID 42496448
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PubMedKidney international2026-07-23

Biallelic pathogenic variants in EXOSC3 mediate renal thrombotic microangiopathy of the kidney.

Walsh Patrick R PR, Basu Uttiya U, Barakat Tahsin Stefan TS, Beck Bodo B BB et al.

Thrombotic microangiopathy (TMA) is characterized by the classical triad of microangiopathic hemolytic anemia, thrombocytopenia and acute kidney injury. Complement inhibition with eculizumab is highly efficacious in TMA secondary to complement dysregulation. However, there are a growing number of eculizumab nonresponsive TMAs reported. Recently a syndromic form of TMA due to recessive variants in RNA exosome components (EXOSC3, EXOSC5) has been identified. The underlying pathogenesis remains unclear. We identified 34 children across Europe with pontocerebellar hypoplasia 1b (PCH1b) due to EXOSC3 rare variants and reviewed their clinical history for signs of TMA. To further examine the pathogenesis, a tamoxifen-inducible whole body Exosc3 conditional knockout mouse model (Exosc3KO) was used. Thirteen (eight male, five female) cases of EXOSC3-TMA were identified. In the United Kingdom the incidence of EXOSC3-TMA was 0.004/million/year. Three children received long-term eculizumab therapy, one child failed to respond and two relapsed on treatment. Exosc3KO demonstrated cell cycle arrest and apoptosis resulting in death in a median of eight days with sequelae noted in actively dividing cells in the bone marrow and large intestine. In this timeframe no kidney pathology was identified. EXOSC3-TMA is a severe, early-onset, C5 inhibitor resistant TMA. EXOSC3-TMA should be considered in eculizumab resistant pediatric TMA, particularly in the context of neurodevelopmental disease.

PMID 42486191
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PubMedBMJ open2026-07-22

Clinical similarity in cost-comparison evaluations: a systematic review of current methods in NICE appraisals and the development of a framework for the formal assessment of clinical similarity.

Edwards Steven J SJ, Burgess Benjamin J BJ, Downes Nicole N, Ip Sophie S et al.

To review how statistically non-significant indirect treatment comparison (ITC) results are interpreted within National Institute for Health and Care Excellence (NICE) cost-comparison evaluations (CCEs) and develop a framework to support interpretations of these results from Bayesian network meta-analyses (NMAs). A systematic review of CCEs between 2017 (first introduced) and April 2025. A framework (point-and-density plots) was developed to better interpret statistically non-significant NMA results for CCEs. CCEs were identified through NICE website searches, references of similar reviews and communications with NICE. NICE technology appraisals (from 2017) that followed a CCE approach ab initio, had final guidance available and used non-statistically significant ITC results were included. A single reviewer performed screening and data extraction with validation by a second reviewer. Narrative syntheses were performed separately for company, External Assessment Group (EAG) and committee perspectives. Point-and-density plots combine elements of forest plots and density plots alongside reporting the probability that a treatment is non-inferior relative to a comparator. These were applied to a recent CCE (TA1019) for crovalimab for patients with paroxysmal nocturnal haemoglobinuria. Among 41 CCEs, EAGs raised concerns about statistically non-significant ITC results while companies relied heavily on them. Only ∼32% of CCEs applied formal methods to explore ITC result uncertainty.For the example framework analysis, comparisons of crovalimab to eculizumab (mean difference (MD): 0.018; 95% CIs -0.22 to 0.25) and ravulizumab (MD: 0.079; 95% CIs -0.25 to 0.41) were statistically non-significant, with non-inferiority not demonstrated. However, point-and-density plots indicated a 95.9% and 86.3% probability of non-inferiority of crovalimab versus eculizumab and ravulizumab. Interpretations of statistically non-significant ITC results are inconsistent within individual CCEs and across appraisals. Implementation of the presented recommendations and framework would improve the consistency and robustness of CCEs. CRD420251034143.

PMID 42481194
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PubMedNephron2026-07-21

Sustained terminal complement inhibition on the endothelium with ravulizumab in patients with atypical Haemolytic Uremic Syndrome.

Gastoldi Sara S, Bresin Elena E, Pasini Andrea A, Nardini Beatrice B et al.

Atypical haemolytic uremic syndrome (aHUS) is a rare and severe form of thrombotic microangiopathy caused by dysregulation of the alternative complement pathway, leading to sustained complement activation at endothelial level and microvascular thrombosis, predominantly affecting the kidney. Anti-C5 therapy with eculizumab has markedly improved patient outcomes, with ex-vivo studies showing suppression of endothelial C5b-9 deposition by sera from treated patients, in contrast to the marked C5b-9 formation induced by acute-phase sera. Ravulizumab, a long-acting anti-C5 antibody derived from eculizumab, has recently been approved for aHUS, offering extended dosing intervals, and maintaining (in patients switched from eculizumab) or inducing (in de novo treated patients) disease remission. Its ability to inhibit terminal complement activation at the endothelial level has not been yet evaluated. In this retrospective study, six patients with primary aHUS were assessed, including four switched from eculizumab and two treated de novo with ravulizumab. Serum-induced C5b-9 formation on human microvascular endothelial cells (HMEC-1) was measured ex-vivo at multiple time points during treatment. C5b-9 formation was in normal range on HMEC-1 exposed to sera collected from all aHUS patients treated with ravulizumab, regardless of prior eculizumab exposure, and all patients remained in or achieved clinical remission. These findings provide mechanistic evidence supporting ravulizumab as an effective inhibitor of terminal complement activation and highlight ex-vivo C5b-9 assessment as a potential tool for monitoring treatment efficacy in aHUS.

PMID 42479640
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PubMedMedicine2026-07-18

Systemic lupus erythematosus complicated by thrombotic microangiopathy with atypical HUS features: A case report.

Lin Yuting Y, Guo Jinjian J

Systemic lupus erythematosus (SLE)-associated thrombotic microangiopathy with features of atypical hemolytic uremic syndrome (aHUS) is a rare but life-threatening complication. Early recognition is challenging because of its overlapping clinical manifestations with other thrombotic microangiopathies. A 49-year-old man with SLE presented with anemia, thrombocytopenia, and multi-system involvement. Despite initial immunosuppressive therapy, his hematologic parameters progressively deteriorated, with hemoglobin decreasing to 89 g/L and the platelet count to 54 × 109/L. Laboratory investigations demonstrated elevated lactate dehydrogenase (501 U/L) and markedly increased soluble complement membrane attack complex (sC5b-9, >1790 ng/mL). ADAMTS13 activity was normal (75.41%), excluding thrombotic thrombocytopenic purpura. Based on the clinical and laboratory findings, the patient was diagnosed with SLE-associated aHUS. Following the diagnosis, the patient received eculizumab, a monoclonal antibody targeting complement component C5. Within 6 days of eculizumab treatment, hemoglobin increased to 102 g/L, and the platelet count recovered to 108 × 109/L, indicating a rapid hematologic response. This case highlights that aHUS can occur as a severe complication of SLE, even in male patients. Complement-mediated thrombotic microangiopathy may coexist with and be triggered by immune hemolysis in SLE. Early recognition and prompt complement inhibition with eculizumab can produce an ultra-rapid hematologic response and may be critical for improving patient outcomes.

PMID 42470044
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PubMedNeurology and therapy2026-07-16

Comparative Evaluation of Rituximab Versus Approved Therapies in Aquaporin-4-IgG-Positive Neuromyelitis Optica Spectrum Disorder: A Systematic Review and Network Meta-analysis.

Barzegar Mahdi M, Samadzadeh Sara S, Audoin Bertrand B, Berthele Achim A et al.

Neuromyelitis optica spectrum disorder (NMOSD) is a rare antibody-mediated neuro-autoimmune disease. Monoclonal antibodies targeting B cell antigens CD19 and CD20, the interleukin-6 receptor, or the complement cascade are used as preventive therapies to reduce relapse rates. We conducted a network meta-analysis (NMA) to compare the effect of rituximab on time to first relapse with ravulizumab, eculizumab, inebilizumab, and satralizumab in patients with NMOSD who are aquaporin-4 (AQP4)-IgG-positive. A systematic search was conducted in PubMed, Scopus, CINAHL, EMBASE, Web of Science, the Cochrane Library, and gray literature sources up to October 31, 2024, and updated on November 1, 2025, following PRISMA guidelines. A network meta-analysis of randomized and open-label trials was conducted to compare time to first relapse between rituximab and other monoclonal antibody therapies. From 6337 records, 3825 duplicates were removed; 2512 were screened, 2327 excluded, leaving eight trials. The prior treatment, relapse history, and definitions and adjudication of relapse varied across studies. Rituximab showed higher hazard ratio (HR) point estimates for time to first relapse compared with ravulizumab with or without immunosuppressive therapies (IST) (HR 5.00, 95% CI 0.25, 101.01) and eculizumab ± IST (HR 1.17, 95% CI 0.12, 10.89), but were lower compared with satralizumab ± IST (HR 0.29, 95% CI 0.04, 2.23). In patients not receiving IST, rituximab showed numerically higher HR compared with ravulizumab (HR 3.33, 95% CI 0.13, 83.16) and eculizumab (HR 1.59, 95% CI 0.05, 50.17), but lower point estimates compared with inebilizumab (HR 0.31, 95% CI 0.04, 2.31) and satralizumab (HR 0.27, 95% CI 0.03, 2.21). This NMA showed hazard ratio point estimates favoring eculizumab and ravulizumab over rituximab. However, wide, overlapping confidence intervals and between-study heterogeneity indicate substantial uncertainty. Head-to-head trials or registry-based studies are needed to determine the most effective treatment for AQP4-IgG-positive NMOSD.

PMID 42458195
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