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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

PubMedClinical case reports2026-10-14

Suboptimal Response to Eculizumab in Renal-Limited Thrombotic Microangiopathy Associated With Malignant Hypertension and Monoclonal Gammopathy: A Case Report.

Lei Wenjuan W, Shi Tianli T, Xu Man M, Zhou Jun J

Renal-limited thrombotic microangiopathy (TMA) is clinically milder but etiologically complex and is often underdiagnosed. This case presents as complement-mediated TMA with malignant hypertension and monoclonal gammopathy but showed suboptimal response to eculizumab treatment, suggesting heterogeneity in both etiology and treatment outcomes.

PMID 42871604
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PubMedFrontiers in immunology2026-10-13

Eculizumab in rapidly progressive glomerulonephritis: a case-series report.

Li Xueting X, Zhang Yinghui Y, Ke Ben B

To describe the clinical outcomes and safety profile of eculizumab as adjunctive therapy in a case series of patients with rapidly progressive glomerulonephritis (RPGN) due to anti-glomerular basement membrane (GBM) disease and anti-neutrophil cytoplasmic antibody-associated vasculitis (AAV). We reported 7 patients with AAV and 5 patients with anti-GBM disease who presented to our center between March 2025 and January 2026. All patients received eculizumab (900 mg, 1-4 doses) in addition to standard immunosuppressive therapy. Serum creatinine (Scr), urinary red blood cell (uRBC) count, urine protein-to-creatinine ratio (UPCR), and dialysis dependence were assessed at baseline and at 3 months. Adverse events were monitored throughout follow-up. In AAV patients, mean Scr decreased from 407.38 μmol/L to 231.27 μmol/L (p = 0.025), mean uRBC from 384.27/μL to 68.75/μL (p = 0.033), and mean UPCR from 2.20 g/g to 1.64 g/g (p = 0.179) at the 3rd month. In anti-GBM patients, mean Scr declined from 575.07 μmol/L to 263.80 μmol/L (p = 0.024), mean uRBC from 1648.60/μL to 461.92/μL (p = 0.132), and mean UPCR from 10.88 g/g to 3.75 g/g (p = 0.126). At baseline, 6 patients required renal replacement therapy, and after treatment, 4 became dialysis-independent. One AAV patient progressed to hemodialysis, and one anti-GBM patient remained dialysis-dependent. All anti-GBM antibodies and MPO-ANCA (except one patient) seroconverted to negative at month 3. During follow-up, only one case of COVID-19 infection and one oral ulcer were recorded; no severe infections occurred. In this descriptive case series, we observed trends toward improved renal function and hematuria at 3 months in patients with severe RPGN who received eculizumab in addition to standard immunosuppression, with earlier initiation and milder baseline renal impairment appearing to correlate with more favorable outcomes. Nevertheless, the lack of a control group and the marked interpatient variability in baseline characteristics and concomitant therapies preclude any conclusion regarding a causal effect of eculizumab. Instead, these data serve as real-world observations that can generate hypotheses for future studies. Definitive evidence on the efficacy and safety of eculizumab in RPGN must await larger, prospective, controlled investigations.

PMID 42865689
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PubMedCase reports in hematology2026-10-13

Excellent Response to Eculizumab in a Patient With Complement-Mediated Thrombotic Microangiopathy and a Novel Plasminogen Gene Variant.

Patel Aman A, Powers Bowen B, Tsai Jason J, Nukala Suresh S

A middle-aged man presented with myalgia, fever, hematemesis, and melena. He was found to have acute kidney injury, acute coronary syndrome, thrombocytopenia, and Coombs-negative hemolytic anemia, raising concern for thrombotic microangiopathy (TMA). While awaiting confirmatory testing, empirical plasma exchange was initiated. Initially, minimal red blood cell fragmentation was observed, which later progressed to florid fragmentation. Despite supportive care, his clinical status deteriorated requiring hemodialysis. The presence of TMA, along with ADAMTS13 metalloprotease activity above the diagnostic threshold, worsening kidney function, and negative stool Shiga toxin testing suggested complement-mediated TMA (C-TMA). Stool PCR testing revealed rotavirus. Eculizumab was initiated, resulting in rapid normalization of hematological parameters and eventual renal recovery. Genetic testing identified a heterozygous PLG c.1735G > A (p.Gly579Arg) variant classified as a variant of uncertain significance (VUS). The importance of serial review of peripheral blood smears and the diagnostic challenge of differentiating TTP, STEC-HUS, and C-TMA are the key learning points.

PMID 42866244
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PubMedClinical case reports2026-10-13

Case Report: Aquaporin-4-Positive Neuromyelitis Optica Spectrum Disorder: Diagnostic Challenges and Transition From B-Cell Depletion Therapy to Ravulizumab.

Zabeti Aram A

This case report illustrates the real-world efficacy of complement component 5 inhibitors in a patient with neuromyelitis optica spectrum disorder who switched from rituximab because of B-cell repopulation occurring early in the course of treatment. Maintenance of remission without relapse was observed when transitioning from rituximab to eculizumab and then to ravulizumab. Results observed in this case support the use of targeted complement inhibition as relapse-preventive therapy after rituximab.

PMID 42865708
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PubMedFrontiers in immunology2026-10-13

Thrombocytopenia: from physiological homeostasis imbalance to mechanism-driven precision diagnosis and therapy.

Pan Yuting Y, Yao Dan D, Le Miaoya M, Tan Zhengwei Z et al.

Thrombocytopenia is not a monolithic disease but a common final pathway of disrupted platelet homeostasis spanning production, clearance, and distribution. The traditional count-centric paradigm, relying on empirical transfusion or steroid administration, frequently obscures underlying mechanistic drivers. This review advances a framework progressing from physiological homeostasis imbalance through pathological mechanisms to precision diagnosis and therapy. Recognizing that immune-targeted therapies are only appropriate when cytopenia is genuinely immune-mediated, we systematically deconstruct both immune and non-immune mechanisms to enable accurate etiological anchoring and avoid inappropriate immunomodulation. We systematically deconstruct the multidimensional pathogenesis along five axes: impaired production, immune-mediated destruction, distributional abnormality, consumptive loss, and pseudothrombocytopenia. Key molecular insights include megakaryocyte transcriptional governance (GATA-1, FLI-1, RUNX1), the platelet desialylation-Ashwell-Morell receptor clearance axis, complement cascade activation, and FcRn-mediated IgG homeostasis. Building on this mechanistic foundation, we propose a three-stage diagnostic algorithm: artifact exclusion, mechanistic localization via non-invasive biomarkers (IPF, TPO, autoantibodies), and etiological anchoring. In the therapeutic landscape, Thrombopoietin receptor agonists (TPO-RAs), FcRn antagonists (efgartigimod and rozanolixizumab), BTK inhibitors (rilzabrutinib), and Syk inhibitors have reshaped ITP management, while complement pathway inhibition (eculizumab, ravulizumab, narsoplimab) has transformed thrombotic microangiopathy outcomes.We posit that thrombocytopenia management has entered the precision medicine era; nevertheless, dynamic biomarker development for refractory cases, artificial intelligence-assisted decision-making, and interdisciplinary integration remain pressing frontiers.

PMID 42866340
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PubMedCEN case reports2026-10-12

Delayed eculizumab therapy guided by markedly elevated soluble C5b-9 in a child with dialysis-requiring acute poststreptococcal glomerulonephritis: a case report.

Xu Ke K, Bai Wei W, Wang Suxia S, Ren Yali Y et al.

Eculizumab, a monoclonal antibody targeting complement C5, has been shown to be effective when administered within 2-4 days of disease onset in children with severe acute poststreptococcal glomerulonephritis (APSGN). However, since APSGN typically follows a self-limited clinical course in most patients, the optimal timing and latest feasible time window for eculizumab initiation in APSGN remain undefined. A 9-year-old girl presented with gross haematuria, oliguria, and renal dysfunction 9 days after the onset of vomiting and abdominal pain. Laboratory tests showed a positive anti-streptolysin O titre, decreased serum complement C3 levels, massive proteinuria, and stage 3 acute kidney injury. Following high-dose methylprednisolone and continuous renal replacement therapy, her serum C3 levels recovered to normal range, yet oliguria and renal impairment persisted two weeks after disease onset. Since serum complement membrane attack complex (C5b-9) remained substantially elevated, eculizumab was initiated at a delayed time point compared with previous reports, yielding a rapid improvement in renal function. Kidney biopsy performed post-treatment with eculizumab revealed focal proliferative glomerulonephritis, accompanied by acute tubular injury. At the 6-month follow-up, except for haematuria detected by dipstick urinalysis, the patient had normal renal function, serum complement C3 levels and negative urinary protein. No severe adverse events occurred. We present a case of APSGN with persistent renal dysfunction and excessive complement activation 2 weeks after glucocorticoid therapy, who achieved substantial clinical remission upon eculizumab administration. Our observation suggests eculizumab may be an effective rescue intervention for persistent renal dysfunction in severe APSGN driven by excessive complement activation.

PMID 42862426
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