Drug Database
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

PubMedFrontiers in medicine2026-05-25

Case Report: Complement-mediated thrombotic microangiopathy masquerading as a pancreatic mass.

Sun Chuanchuan C, Ding Ying Y, Yang Fan F, Ding Yunfei Y et al.

Complement-mediated thrombotic microangiopathy (CM-TMA) results from dysregulated alternative pathway activation. Although extra-renal manifestations are well-recognized, presentation as a pancreatic mass is exceedingly rare and risks misdiagnosis as malignancy, delaying critical intervention. A 28-year-old male presented with malignant hypertension and rapidly progressive renal impairment. Abdominal CT revealed a pancreatic tail mass suspicious for neoplasia. However, histopathology of both renal and pancreatic tissues demonstrated microvascular thrombosis and endothelial injury, pointing to a systemic TMA process. Subsequent genetic testing confirmed pathogenic variants in CFH (p.Tyr1058His, p.Val1060Leu) and THBD (p.Asp486Tyr), establishing the diagnosis of CM-TMA. Treatment was initiated with eculizumab alongside intensive renin-angiotensin system blockade. After 6 months of therapy, renal function recovered and the pancreatic mass completely resolved. However, 3 months after self-discontinuing eculizumab, the patient experienced a severe disease relapse and subsequently entered maintenance hemodialysis. This first histopathologically confirmed case of adult CM-TMA presenting as a pancreatic mass expands the known phenotype of complement-mediated disease. It highlights that in young adults with unexplained malignant hypertension and multisystem involvement, prompt evaluation for complement abnormalities is critical. The presence of high-risk complement variants and the relapse following treatment cessation underscore the necessity for long-term complement inhibition in such patients.

PMID 42180719
Read full article →
PubMedFrontiers in medicine2026-05-25

Paroxysmal nocturnal hemoglobinuria with a positive Coombs test presenting as acute kidney injury: a case report.

Jiang Luyao L, Chen Tianxi T, Xu Linlin L, Shi Miaojun M et al.

This report presents the case of a 60-year-old woman who was admitted with symptoms of fatigue and poor appetite. Laboratory investigations revealed severe acute kidney injury (AKI), indicated by a serum creatinine level of 2,065 μmol/L, necessitating the initiation of emergency hemodialysis. The patient also exhibited hemolytic anemia (hemoglobin, 69 g/L), thrombocytopenia (platelet nadir, 45 × 109/L), venous thrombosis, a positive direct antiglobulin test (DAT), and a low absolute reticulocyte count. Conventional flow cytometry (red blood cells and neutrophils CD55/CD59) did not detect a paroxysmal nocturnal hemoglobinuria (PNH) clone. Renal biopsy revealed hemoglobin cast nephropathy and intrarenal venous thrombosis. Subsequent bone marrow evaluation, high-sensitivity flow cytometry (FLAER method), and supportive PIGA sequencing findings established the diagnosis of PNH. Treatment with eculizumab led to hematologic remission and progressive renal recovery. This case underscores the importance of considering PNH in patients with severe AKI and hemolysis, even when initial conventional flow cytometry is negative.

PMID 42180722
Read full article →
PubMedNephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association2026-05-24

Infection risk mitigation with complement inhibitors in kidney disease.

Berghmans Mathilde M, Seghers François F, Mirioglu Safak S, Kronbichler Andreas A et al.

Complement inhibitors are increasingly used in kidney diseases, ranging from complement-mediated thrombotic microangiopathies to glomerulonephritis. Because complement is central to host defence against encapsulated bacteria, therapeutic blockade creates a predictable infectious vulnerability requiring structured prevention strategies. This narrative review integrates complement biology with evidence from pivotal kidney trials, pharmacovigilance analyses, and national public health recommendations to propose a pragmatic framework for mitigating infection-risk in patients treated with complement inhibitors. Key complement functions (C3b-mediated opsonisation, C3a/C5a-driven inflammation, and membrane attack complex formation) underlie target-specific patterns of infectious risk. Terminal C5 inhibition (eculizumab, ravulizumab) confers a reproducible exposure-adjusted risk of invasive meningococcal disease, including rare but documented infection-related mortality, with breakthrough infections reported despite vaccination. In contrast, inhibition at the level of C3/C3b or the alternative pathway amplification loop has not, to date, demonstrated a consistent encapsulated-pathogen signal in phase 3 kidney trials, although follow-up remains limited and rare events cannot be excluded. Effective prevention relies on target-adapted vaccination against encapsulated bacteria, consideration of antimicrobial prophylaxis when therapy must begin before vaccine protection is established-and in selected higher-risk settings during sustained complement blockade-and structured patient education with rapid-access pathways for febrile illness. Chronic kidney disease, dialysis exposure, transplantation, and concomitant immunosuppression may further amplify infectious risk and attenuate vaccine responsiveness. Infection risk under complement inhibition is target-dependent and not eliminated by vaccination alone, particularly with terminal C5 blockade. Mechanism-informed, kidney-specific prevention strategies are essential as complement therapies expand into routine clinical practice.

PMID 42175904
Read full article →
PubMedNeurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology2026-05-22

Anti-acetylcholine receptor antibody overshoot following efgartigimod in myasthenia gravis: Two case reports with literature review.

Yang Rui-Yuan RY, Wang Bian-Rong BR, Hong Ye Y, Fu Yi-Jia YJ et al.

FcRn antagonists have reshaped generalized myasthenia gravis (MG) treatment by enhancing IgG catabolism, complementing traditional antibody-depleting therapy (ADT). However, disrupting IgG homeostasis may paradoxically cause antibody overshoot, potentially worsening MG symptoms. The risk factors and optimal management of antibody overshoot remain inadequately characterized. We report two novel cases of anti-acetylcholine receptor (AChR) antibody overshoot and clinical exacerbation following FcRn antagonist efgartigimod treatment. To contextualize these findings, we performed a literature search in PubMed, identifying 10 previously reported cases of antibody overshoot in MG (2 following efgartigimod and 8 following plasma exchange). In the efgartigimod cohort (n=4, including our two novel cases), all patients were anti AChR positive, had thymoma associated MG (TAMG, 100%), and had undergone prior thymectomy (100%). Notably, none had received adequate background immunosuppressive therapy before efgartigimod initiation (75% inadequate, 25% none). All patients (100%) experienced severe clinical exacerbation (MGFA class V in the 3 patients with available data). Rescue therapies-including corticosteroids (75%), eculizumab (50%), IVIG (50%), and tacrolimus (50%)-were effective, with all four patients showing significant improvement. Furthermore, analysis of the plasma exchange cohort (n=8) and the combined cohort (n=12) revealed a distinct and shared clinical risk profile for the development of antibody overshoot. Anti AChR antibody overshoot following efgartigimod therapy is a rare but clinically significant adverse event, predominantly affecting patients with TAMG or prior thymectomy who lack adequate baseline immunosuppression. Given the dissociation between total IgG reduction and autoantibody rebound, monitoring should focus specifically on anti-AChR titer rather than total IgG level alone.

PMID 42168668
Read full article →
PubMedKidney international reports2026-05-21

Living Donor Kidney Transplantation in High-Recurrence Kidney Diseases: Precision Risk Stratification and Management.

Verhellen Catherine C, Devresse Arnaud A, Gillion Valentine V, Van Regemorter Elliott E et al.

Living-donor kidney transplantation (KT) offers the best outcomes for patients with kidney failure (KF). However, recurrence of primary kidney disease in the allograft remains a major concern and represents 1 of the leading causes of graft loss, significantly influencing donor and recipient selection. The recurrence risk varies substantially among kidney diseases and must be carefully assessed during pretransplant evaluation. For diseases associated with low-to-intermediate recurrence risk, such as lupus nephritis, antineutrophil cytoplasmic antibodies-associated vasculitis, or IgA nephropathy, living-donor KT is generally safe, although appropriate counseling remains essential. In contrast, diseases such as membranous nephropathy (MN), primary focal segmental glomerulosclerosis (FSGS), atypical hemolytic uremic syndrome (aHUS), C3 glomerulopathy (C3G), primary hyperoxaluria (PH), and amyloid light-chain (AL) amyloidosis are associated with higher recurrence rates and often require complex, individualized assessment. Recent advances have significantly transformed this field. Biomarkers such as anti-phospholipase A2 receptor (anti-PLA2R) antibodies in MN and antinephrin antibodies in FSGS enable refined risk stratification and tailored pre- and post-transplant strategies. Complement gene analysis and targeted therapies, including eculizumab, have markedly improved outcomes in aHUS, whereas novel complement inhibitors show promise for recurrent C3G. RNA interference therapy has expanded kidney-alone transplantation options in PH, and modern hematologic therapies have improved post-transplant prognosis in AL amyloidosis. These developments are reshaping the decision-making landscape for living-donor KT in diseases with a high risk of recurrence, highlighting the need for multidisciplinary evaluation, individualized strategies, and transparent donor-recipient communication to optimize transplant outcomes.

PMID 42164636
Read full article →
PubMedMolecular biology reports2026-05-14

A review of genetic and epigenetic biomarkers involved in the occurrence of atypical hemolytic uremic syndrome and its therapeutic strategies.

Tohidi Mohamadreza M, Mohammadi Mahan M

Atypical hemolytic uremic syndrome (aHUS) is a rare, chronic, and life-threatening thrombotic microangiopathy (TMA). The disease is most frequently linked to genetic or acquired dysregulation of the alternative complement pathway and affects both children and adults. The paucity of a gold standard for diagnostic testing and the variety of clinical manifestations are the primary reasons for the challenge in accurately diagnosing aHUS, which can have an impact on patient care. This is due to the fact that the more prevalent autoinflammatory diseases are multifactorial in nature, with both genetic and non-genetic factors playing critical roles. Until the last decade, treatment options were limited to plasma therapy and, in selected cases, liver transplantation, neither of which directly addressed the underlying pathophysiology and both of which carried substantial risks. Since its introduction in 2011, the anti-C5 monoclonal antibody eculizumab has significantly improved outcomes and transformed disease management. Trials on novel complement inhibitors, regenerative medicine, targeted therapy, and stem cell therapy may also improve future treatment options. The primary objectives of this investigation are to identify the genetic and epigenetic causes of aHUS, as well as the limitations and debates surrounding its management. Due to the prevalence and significance of this type of illness, researchers have investigated effective therapeutic methods and more recent approaches.

PMID 42133203
Read full article →

+3013 more articles available with a free account

Sign up free to view all articles →

Ask about eculizumab