Ravulizumab

Summary

Ravulizumab (CHEMBL3989986) is an approved antibody (ATC L04AJ02); indicated across 13 conditions including hemolytic-uremic syndrome and hemoglobinuria.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Antibody
• ATC class: L04AJ02
• Indications: 13 conditions
• Clinical trials: 50

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3989986
Name	Ravulizumab
Type	Antibody
Max phase	4
ATC	L04AJ02

Also known as: ALXN-1210, ALXN1210, Ravulizumab, Ravulizumab cwvz, Ravulizumab-cwvz, Ultomiris, RAVULIZUMAB

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

13 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
hemolytic-uremic syndrome	4	MONDO:0001549	MONDO:0001549
hemoglobinuria	4	MONDO:0003656	MONDO:0100244
myasthenia gravis	3	MONDO:0009688	EFO:0004991
neuromyelitis optica	3	MONDO:0019100	EFO:0004256
acute lung injury	3	MONDO:0015796	EFO:0004610
viral pneumonia	3	MONDO:0006012	EFO:0007541
acute respiratory distress syndrome	3	MONDO:0006502	EFO:1000637
amyotrophic lateral sclerosis	3	MONDO:0004976	MONDO:0004976
atypical hemolytic-uremic syndrome	3	MONDO:0016244	MONDO:0016244
thrombotic microangiopathy	3	MONDO:0019737	MONDO:0019737
IgA glomerulonephritis	3	MONDO:0005342	EFO:0004194
dermatomyositis	2	MONDO:0016367	EFO:0000398
preeclampsia	2	MONDO:0005081	EFO:0000668

Clinical trials

Total trials: 50.

Phase distribution

Phase	Trials
PHASE3	25
Not specified	8
PHASE4	5
PHASE2	5
PHASE1	4
PHASE2/PHASE3	2
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07010302	PHASE4	NOT_YET_RECRUITING	Rituximab Versus Ravulizumab, Inebilizumab, Satralizumab, and Eculizumab in NMOSD
NCT07221838	PHASE4	RECRUITING	A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab
NCT07308574	PHASE4	RECRUITING	Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS
NCT04320602	PHASE4	COMPLETED	Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab
NCT04390464	PHASE4	UNKNOWN	mulTi-Arm Therapeutic Study in Pre-ICu Patients Admitted With Covid-19 - Repurposed Drugs (TACTIC-R)
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
NCT05346354	PHASE2/PHASE3	RECRUITING	Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD
NCT05644561	PHASE3	ACTIVE_NOT_RECRUITING	Evaluation of PK, PD, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants With Generalized Myasthenia Gravis
NCT05746559	PHASE3	ACTIVE_NOT_RECRUITING	ARTEMIS: Ravulizumab to Protect Patients With CKD From CSA-AKI and MAKE
NCT06291376	PHASE3	RECRUITING	Study of Ravulizumab in Immunoglobulin A Nephropathy (IgAN)
NCT06830798	PHASE3	RECRUITING	Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab Administered Intravenously in Adult Participants at High Risk of Delayed Graft Function After Kidney Transplantation
NCT07024563	PHASE3	RECRUITING	Study of Ravulizumab in Pediatric Participants With Primary IgAN
NCT07557420	PHASE3	NOT_YET_RECRUITING	Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity Study of Ravulizumab in Chinese Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD)
NCT07596784	PHASE3	NOT_YET_RECRUITING	Efficacy and Safety of Ravulizumab in Chinese Adults Participants With Generalized Myasthenia Gravis (gMG)
NCT02946463	PHASE3	COMPLETED	ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT02949128	PHASE3	COMPLETED	Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
NCT03056040	PHASE3	COMPLETED	ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
NCT03131219	PHASE3	COMPLETED	Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
NCT03406507	PHASE3	COMPLETED	A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria
NCT03748823	PHASE3	COMPLETED	Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
NCT03920293	PHASE3	COMPLETED	Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis
NCT04201262	PHASE3	COMPLETED	An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD
NCT04248465	PHASE3	TERMINATED	An Efficacy and Safety Study of Ravulizumab in ALS Participants
NCT04369469	PHASE3	TERMINATED	Efficacy and Safety Study of IV Ravulizumab in Patients With COVID-19 Severe Pneumonia
NCT04543591	PHASE3	COMPLETED	Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant
NCT04557735	PHASE3	COMPLETED	Study of Ravulizumab in Pediatric Participants With HSCT-TMA
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
NCT04570397	PHASE3	UNKNOWN	Ravulizumab and COVID-19
NCT04743804	PHASE3	TERMINATED	Ravulizumab in Thrombotic Microangiopathy Associated With a Trigger
NCT04999020	PHASE2/PHASE3	TERMINATED	Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis
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
NCT06578949	PHASE3	COMPLETED	Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT06333652	PHASE2	RECRUITING	Ravulizumab in Pregnancies Complicated by Severe Hypertensive Disorders
NCT02598583	PHASE1/PHASE2	COMPLETED	Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT02605993	PHASE2	COMPLETED	Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT04564339	PHASE2	TERMINATED	Study of Ravulizumab in Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN)
NCT04702568	PHASE2	TERMINATED	A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT05116774	PHASE2	TERMINATED	BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy
NCT05288660	PHASE1	COMPLETED	A Study of a Single Dose of ALXN1210 in Healthy Participants
NCT05288673	PHASE1	COMPLETED	A Study of Multiple Doses of ALXN1210 in Healthy Adult Participants

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).