Back to discover
Drug

Ravulizumab

Approved · EMA
Emerging researchLate-stage development

Also known as Ultomiris.

RxNorm2107301UNIIC3VX249T6L
2
Research papers
12
Active clinical trials
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

Phase 3, Open-label, Single-arm, Multicenter Study Evaluating Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants (6 to <18 Years) With Generalized Myasthenia Gravis (gMG)

Clinical trial2026-07-15Primary completion · ClinicalTrials.gov

A Comparative Clinical Effectiveness Trial of Rituximab Versus Ravulizumab, Inebilizumab, Satralizumab and Eculizumab To Prevent Relapses in Neuromyelitis Optica Spectrum Disorder

Clinical trial2026-06-24Results expected Q1 2030 · ClinicalTrials.gov

A Phase 2/3, Open-label, Historical-controlled, Single-arm, Multicenter Study to Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, and Safety of Ravulizumab in Children and Adolescents With Aquaporin-4 Antibody Positive (AQP4-Ab [+]) Neuromyelitis Optica Spectrum Disorder (NMOSD)

Clinical trial2026-06-22Primary completion · ClinicalTrials.gov

An Open-label, Single-arm, Multi-center, Interventional Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adult Patients With Generalized Myasthenia Gravis (gMG)

Clinical trial2026-06-22Results expected Q3 2027 · ClinicalTrials.gov

A Phase 3, 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

Clinical trial2026-06-18Results expected Q4 2027 · ClinicalTrials.gov

A Phase 3b, Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adult Participants With Neuromyelitis Optica Spectrum Disorder (NMOSD)

Clinical trial2026-06-18Results expected Q4 2028 · ClinicalTrials.gov

A Phase 3, Single-arm, Open-label, Multicenter Study to Assess the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Complement Inhibitor Treatment Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

Clinical trial2026-06-05Results posted · ClinicalTrials.gov

ARTEMIS: RAvulizumab to PRotect PaTients With Chronic Kidney DisEase (CKD) froM Cardiac Surgery Associated Acute Kidney Injury (CSA-AKI) and Subsequent Major Adverse Kidney Events (MAKE): A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study

Clinical trial2026-05-07Completed · ClinicalTrials.gov

A Randomized, Open-Label, C5 Inhibitor-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy

Clinical trial2026-04-30Results expected Q4 2026 · ClinicalTrials.gov

Long-Term Efficacy and Safety of the Long-Acting Complement C5 Inhibitor Ravulizumab for the Treatment of Atypical Hemolytic Uremic Syndrome in Adults.

Research2021-03-24Kidney international reports

The long-acting C5 inhibitor, Ravulizumab, is effective and safe in adult patients with atypical hemolytic uremic syndrome naïve to complement inhibitor treatment.

Research2020-03-06Kidney international

Approval: Ultomiris (EMA)

Regulatory2019-07-02EMA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Ravulizumab
Aliases & brands
Ultomiris
RxNorm CUI
2107301
UNII
C3VX249T6L
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2019-07-02
Latest approval
2019-07-02
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2019-07-02Approval
Approval: Ultomiris (EMA)
Indication: Paroxysmal nocturnal haemoglobinuria (PNH)Ultomiris is indicated in the treatment of adult and paediatric patients with a body weight of 10 kg or above with PNH:- in patients with… Show full indication

Paroxysmal nocturnal haemoglobinuria (PNH)Ultomiris is indicated in the treatment of adult and paediatric patients with a body weight of 10 kg or above with PNH:- in patients with haemolysis with clinical symptom(s) indicative of high disease activity.- in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months (see section 5.1). Atypical haemolytic uremic syndrome (aHUS)Ultomiris is indicated in the treatment of patients with a body weight of 10 kg or above with aHUS who are complement inhibitor treatment-naïve or have received eculizumab for at least 3 months and have evidence of response to eculizumab (see section 5.1). Generalized myasthenia gravis (gMG)Ultomiris is indicated as an add-on to standard therapy for the treatment of adult patients with gMG who are anti-acetylcholine receptor (AChR) antibody-positive. Neuromyelitis Optica Spectrum Disorder (NMOSD)Ultomiris is indicated in the treatment of adult patients with NMOSD who are anti-aquaporin 4 (AQP4) antibody-positive (see section 5.1). Ultomiris is indicated in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH):- in patients with haemolysis with clinical symptom(s) indicative of high disease activity.- in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months. Ultomiris is indicated in the treatment of adult patients with atypical haemolytic uremic syndrome (aHUS) who are complement inhibitor treatment-naïve or have received eculizumab for at least 3 months and have evidence of response to eculizumab.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

38 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
38
registered trials across all phases
LATEST COMPLETION 2026
12
Active studies
8
Recruiting
32
Late-stage (III+)
16
Completed
10
Discontinued
PHASE DISTRIBUTIONn = 38
Phase 11Phase 25Phase 2 / 32Phase 325Phase 45

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Comparative Clinical Effectiveness Trial of Rituximab Versus Ravulizumab, Inebilizumab, Satralizumab and Eculizumab To Prevent Relapses in Neuromyelitis Optica Spectrum Disorder

Not yet recruitingPH IVMassachusetts General Hospitaln=5402030

OCTAGON: A Phase 4, Single Arm Study to Investigate Oral Corticosteroid Tapering in Adult Patients With Generalized Myasthenia Gravis Treated With Ravulizumab

RecruitingPH IVAlexion Pharmaceuticals, Inc.n=752027

Multicenter, Open-label, Single-arm, Post-Marketing Clinical Study to Evaluate the Efficacy and Safety of Ravulizumab in Participants Clinically Diagnosed as Atypical Hemolytic Uremic Syndrome

RecruitingPH IVAlexion Pharmaceuticals, Inc.n=202027

Phase 4, Single-Arm Study of Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab

CompletedPH IVAlexion Pharmaceuticals, Inc.n=182022

mulTi-Arm Therapeutic Study in Pre-ICu Patients Admitted With Covid-19 - Repurposed Drugs (TACTIC-R)

Unknown statusPH IVKey trialCambridge University Hospitals NHS Foundation Trustn=1,1672022

Recent completions

Trials that read out recently, adding to the completed evidence base.

ARTEMIS: RAvulizumab to PRotect PaTients With Chronic Kidney DisEase (CKD) froM Cardiac Surgery Associated Acute Kidney Injury (CSA-AKI) and Subsequent Major Adverse Kidney Events (MAKE): A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study

CompletedPH IIIAlexion Pharmaceuticals, Inc.n=5442026

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Ravulizumab in Adult and Adolescent Participants Who Have Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplant (HSCT)

CompletedPH IIIAlexion Pharmaceuticals, Inc.n=1482026

A Phase 3, Single-arm, Open-label, Multicenter Study to Assess the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Complement Inhibitor Treatment Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

CompletedPH IIIAlexion Pharmaceuticals, Inc.n=182025

A Phase 3, Open-label, Single Arm, Multicenter Study of Ravulizumab in Addition to Best Supportive Care in Pediatric Participants With Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplantation (HSCT)

CompletedPH IIIAlexion Pharmaceuticals, Inc.n=412025

A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD)

CompletedPH IIIAlexion Pharmaceuticals, Inc.n=582024

A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab

CompletedPH IIIAlexion Pharmaceuticals, Inc.n=1392023

Research activity

2 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Publications over time
20202021
Major research themes2
Atypical Hemolytic Uremic Syndrome1Thrombotic Microangiopathies1
Journals, researchers & institutions
Top journals
  • Kidney international1
  • Kidney international reports1
Leading researchers
  • Ariceta G2
  • Barbour T2
  • Cataland S2
  • Heyne N2
  • Kavanagh D2
  • Miyakawa Y2
  • Scully M2
  • Yoon SS2
Leading institutions
free-text, unnormalised
  • Clinical Development2
  • National Renal Complement Therapeutics Centre2
  • Saitama Medical University2
  • Seoul National University Hospital2
  • Sorbonne Université2
  • The Ohio State University Medical Center2
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.