Eculizumab

Summary

Eculizumab (CHEMBL1201828) is an approved antibody (ATC L04AJ01); indicated across 20 conditions including atypical hemolytic-uremic syndrome and hemoglobinuria.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Antibody
• ATC class: L04AJ01
• Indications: 20 conditions
• Clinical trials: 101

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1201828
Name	Eculizumab
Type	Antibody
Max phase	4
ATC	L04AJ01

Also known as: 5G1.1, ABP-959, Alexion, Bekemv, Bkemv, Eculizumab, Eculizumab biosimilar (abp-959), Eculizumab biosimilar (amgen), Eculizumab-aagh, Eculizumab-aeeb, Elizaria, Epysqli

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

20 indications (2 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
atypical hemolytic-uremic syndrome	4	MONDO:0016244	MONDO:0016244
hemoglobinuria	4	MONDO:0003656	MONDO:0100244
myasthenia gravis	3	MONDO:0009688	EFO:0004991
neuromyelitis optica	3	MONDO:0019100	EFO:0004256
leukemia	3	MONDO:0005059	EFO:0000565
Guillain-Barre syndrome	3	MONDO:0016218	EFO:0007292
hemolytic-uremic syndrome	3	MONDO:0001549	MONDO:0001549
anti-neutrophil antibody associated vasculitis	2	MONDO:0005435	EFO:0004826
age-related macular degeneration	2	MONDO:0005150	EFO:0001365
chronic kidney disease	2	MONDO:0005300	EFO:0003884
diabetes mellitus	2	MONDO:0005015	EFO:0000400
thrombocytopenia	2	MONDO:0002049	HP:0001873
dermatomyositis	2	MONDO:0016367	EFO:0000398
severe acute respiratory syndrome	2	MONDO:0005091	MONDO:0100096
membranoproliferative glomerulonephritis	2	MONDO:0002461	MONDO:0018904
autoimmune hemolytic anemia	2	MONDO:0020108	MONDO:0018922
graft versus host disease	1	MONDO:0013730	EFO:0004599

3 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 101.

Phase distribution

Phase	Trials
PHASE3	37
PHASE2	34
Not specified	9
PHASE1/PHASE2	7
PHASE4	5
PHASE1	5
PHASE2/PHASE3	3
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07010302	PHASE4	NOT_YET_RECRUITING	Rituximab Versus Ravulizumab, Inebilizumab, Satralizumab, and Eculizumab in NMOSD
NCT07410039	PHASE4	RECRUITING	Therapy in the Acute Phase of NMOSD: A Multicenter Prospective Real-World Study
NCT00867932	PHASE4	COMPLETED	Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT02574403	PHASE4	COMPLETED	Study Assessing an Algorithm-based Strategy of Eculizumab Discontinuation in Children and Adults With aHUS
NCT04320602	PHASE4	COMPLETED	Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab
NCT04432584	PHASE3	ACTIVE_NOT_RECRUITING	A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
NCT04434092	PHASE3	ACTIVE_NOT_RECRUITING	A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
NCT05133531	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment
NCT06724809	PHASE3	ACTIVE_NOT_RECRUITING	Efficacy, Safety, PK, PD, and ADA of Eculizumab in Chinese Adults With NMOSD
NCT06932744	PHASE3	RECRUITING	Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy
NCT07177859	PHASE3	NOT_YET_RECRUITING	A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients
NCT07420296	PHASE3	RECRUITING	Modified Zipper Therapy for AQP4-IgG Positive Neuromyelitis Optica Spectrum Disorder
NCT00098280	PHASE3	COMPLETED	Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria
NCT00112983	PHASE3	COMPLETED	Eculizumab in Treating Patients With Paroxysmal Nocturnal Hemoglobinuria
NCT00122304	PHASE3	COMPLETED	Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab
NCT00122317	PHASE3	COMPLETED	Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT00122330	PHASE3	COMPLETED	Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
NCT00130000	PHASE3	COMPLETED	Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria
NCT01410916	PHASE2/PHASE3	COMPLETED	Safety and Efficacy Study of Eculizumab in Shiga-Toxin Producing Escherichia Coli Hemolytic-Uremic Syndrome (STEC-HUS)
NCT01892345	PHASE3	TERMINATED	A Randomized Controlled Trial of Eculizumab in AQP4 Antibody-positive Participants With NMO (PREVENT Study)
NCT01997229	PHASE3	COMPLETED	Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)
NCT02003144	PHASE3	COMPLETED	An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients
NCT02013037	PHASE3	COMPLETED	The De-novo Use of Eculizumab in Presensitized Patients Receiving Cardiac Transplantation
NCT02145182	PHASE2/PHASE3	COMPLETED	Prevention of Delayed Graft Function Using Eculizumab Therapy (PROTECT Study)
NCT02205541	PHASE3	COMPLETED	Eculizumab in Shiga-toxin Related Hemolytic and Uremic Syndrome Pediatric Patients - ECULISHU
NCT02301624	PHASE3	COMPLETED	Extension Study of ECU-MG-301 to Evaluate Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis
NCT02946463	PHASE3	COMPLETED	ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT03056040	PHASE3	COMPLETED	ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
NCT03500549	PHASE3	COMPLETED	Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT03759366	PHASE3	COMPLETED	A Phase 3 Open-Label Study of Eculizumab in Pediatric Participants With Refractory Generalized Myasthenia Gravis (gMG)
NCT03818607	PHASE3	COMPLETED	A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH
NCT04058158	PHASE3	COMPLETED	A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria
NCT04060264	PHASE3	COMPLETED	Clinical Trial of BCD-148 and Soliris® for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria
NCT04155424	PHASE2/PHASE3	TERMINATED	A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Spectrum Disorder
NCT04558918	PHASE3	COMPLETED	Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment
NCT04752566	PHASE3	COMPLETED	A Study to Evaluate the Efficacy and Safety of Eculizumab in Guillain-Barré Syndrome
NCT04935177	PHASE3	COMPLETED	Renal Function in Highly Sensitized Patients 1 Year After Desensitization With Imlifidase Prior to DD Kidney Tx
NCT05131204	PHASE3	TERMINATED	Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
NCT05702996	PHASE3	UNKNOWN	Multicenter, Uncontrolled Pilot Study Evaluating the Efficacy of Eculizumab in the Treatment of Gemcitabine-induced Thrombotic Microangiopathies
NCT05876351	PHASE3	COMPLETED	Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).