Ultomiris

(Ravulizumab)

ApprovedMonoclonal antibody

Ultomiris is a monoclonal antibody that works as a Complement C5 Inhibitor.[2] It is being used across 24 indications, with its most advanced program in Approved.[1]

Ultomiris is one of 450 small molecule programs targeting Atypical Hemolytic-Uremic Syndrome. It is currently in Approved.

Indications

Total Trials

Active Trials

449

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Pipeline

Status by Indication[3][1]

Indication

Atypical Hemolytic-Uremic SyndromeActive, not recruiting

IGA GlomerulonephritisActive, not recruiting

Kidney TransplantActive, not recruiting

Viral PneumoniaActive, not recruiting

PneumoniaActive, not recruiting

Acute Lung InjuryActive, not recruiting

Amyotrophic Lateral SclerosisActive, not recruiting

AnemiaActive, not recruiting

Hemolytic-Uremic SyndromeUnknown

Thrombotic MicroangiopathyActive, not recruiting

Atypical Hemolytic-Uremic SyndromePhase 3

Active, not recruiting

IGA GlomerulonephritisPhase 3

Active, not recruiting

Kidney TransplantPhase 3

Active, not recruiting

Viral PneumoniaPhase 3

Active, not recruiting

PneumoniaPhase 3

Active, not recruiting

Acute Lung InjuryPhase 3

Active, not recruiting

Amyotrophic Lateral SclerosisPhase 3

Active, not recruiting

AnemiaPhase 3

Active, not recruiting

Hemolytic-Uremic SyndromePreclinical

Unknown

Thrombotic MicroangiopathyPhase 3

Active, not recruiting

Showing 10 of 24 indications

Competition

Competitive Landscape

1931 competitors in COVID-19

#DrugCompanyPhaseTrials

Ultomiris

AstraZeneca

Approved

2 2

Bnt162b2

BioNTech

Preclinical

29 3

Aldactone

Pfizer

Preclinical

1 4

Stivarga

Bayer

Preclinical

1 5

Aimovig

Amgen

Preclinical

1 6

Singulair

AstraZeneca

Preclinical

1 7

Calciferol

Alnylam Pharmaceuticals

Activella

Nitrostat

VViatris Specialty LLC

Preclinical

1 10

SARS-COV-2 VACCINE

Moderna

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q1 2027

Phase 3Recruiting

Paroxysmal Nocturnal Hemoglobinuria

NCT05133531

Q2 2027

Post-ApprovalRecruiting

ART

NCT07308574

Recruiting

Neuromyelitis Optica

NCT06398158

Recruiting

Myasthenia Gravis

NCT06967480

Q3 2027

Post-ApprovalNot yet recruiting

Myasthenia Gravis

NCT07221838

Phase 3Not yet recruiting

Generalized Myasthenia Gravis

NCT07596784

Clinical

Trials by Indication[1]

Paroxysmal Nocturnal Hemoglobinuria12 trials

Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

Completed· AstraZeneca logo

AstraZeneca·Phase 3·NCT03748823·139 enrolled

BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy

Terminated·BBioCryst Pharmaceuticals·Phase 2·NCT05116774·12 enrolled

Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria

Completed· AstraZeneca logo

AstraZeneca··NCT05274633·120 enrolled

Show 9 more trials

ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

Completed· AstraZeneca logo

AstraZeneca·Phase 3·NCT03056040·202 enrolled

ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Completed· AstraZeneca logo

AstraZeneca·Phase 3·NCT02946463·272 enrolled

A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria

Completed· AstraZeneca logo

AstraZeneca·Phase 3·NCT03406507·13 enrolled

Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Completed· AstraZeneca logo

AstraZeneca·Phase 2·NCT02605993·26 enrolled

Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Completed· AstraZeneca logo

AstraZeneca·Phase 3·NCT06578949·18 enrolled

Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab

Completed· AstraZeneca logo

AstraZeneca·Phase 4·NCT04320602·18 enrolled

A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Terminated·BBioCryst Pharmaceuticals·Phase 2·NCT04702568·19 enrolled

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

Recruiting· Regeneron logo

Regeneron·Phase 3·NCT05133531·190 enrolled

Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria

Terminated· Regeneron logo

Regeneron·Phase 3·NCT05131204·3 enrolled

Myasthenia Gravis5 trials

Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)

Active, not recruiting· AstraZeneca logo

AstraZeneca·Phase 3·NCT05644561·12 enrolled

Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis

Completed· AstraZeneca logo

AstraZeneca·Phase 3·NCT03920293·175 enrolled

A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab

Not yet recruiting· AstraZeneca logo

AstraZeneca·Phase 4·NCT07221838·75 enrolled

Show 2 more trials

Ravulizumab Treatment Outcomes in Patients With Generalized Myasthenia Gravis

Recruiting· AstraZeneca logo

AstraZeneca··NCT06909253·50 enrolled

Early Ravulizumab Treatment Of Anti- AChR Antibody-Positive Generalized Myasthenia Gravis

Recruiting· AstraZeneca logo

AstraZeneca··NCT06967480·40 enrolled

Atypical Hemolytic-Uremic Syndrome3 trials

Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)

Completed· AstraZeneca logo

AstraZeneca·Phase 3·NCT03131219·34 enrolled

Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

Completed· AstraZeneca logo

AstraZeneca·Phase 3·NCT02949128·58 enrolled

A Non-interventional Study Evaluating Ravulizumab Treatment Outcomes in Polish Patients With Atypical Hemolytic Uremic Syndrome

Recruiting· AstraZeneca logo

AstraZeneca··NCT07399730·80 enrolled

Thrombotic Microangiopathy2 trials

Ravulizumab in Thrombotic Microangiopathy Associated With a Trigger

Terminated· AstraZeneca logo

AstraZeneca·Phase 3·NCT04743804·16 enrolled

Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant

Active, not recruiting· AstraZeneca logo

AstraZeneca·Phase 3·NCT04543591·148 enrolled

Pediatric2 trials

Study of Ravulizumab in Pediatric Participants With HSCT-TMA

Completed· AstraZeneca logo

AstraZeneca·Phase 3·NCT04557735·41 enrolled

Study of Ravulizumab in Pediatric Participants With Primary IgAN

Recruiting· AstraZeneca logo

AstraZeneca·Phase 3·NCT07024563·24 enrolled

17 more indications

COVID-192 trials

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

Unknown status·—·Phase 4·NCT04390464·1,167 enrolled

Ravulizumab and COVID-19

Unknown status·—·Phase 3·NCT04570397·32 enrolled

Efficacy and Safety2 trials

An Efficacy and Safety Study of Ravulizumab in ALS Participants

Terminated· AstraZeneca logo

AstraZeneca·Phase 3·NCT04248465·382 enrolled

An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD

Completed· AstraZeneca logo

AstraZeneca·Phase 3·NCT04201262·58 enrolled

Treatment Outcome1 trial

Real-life Treatment Outcomes of Ravulizumab in PNH

Active, not recruiting· AstraZeneca logo

AstraZeneca··NCT06633536·64 enrolled

Anemia1 trial

Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment

Completed· Novartis logo

Novartis·Phase 3·NCT04558918·97 enrolled

ART1 trial

Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS

Recruiting· AstraZeneca logo

AstraZeneca·Phase 4·NCT07308574·20 enrolled

Dermatomyositis1 trial

Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis

Terminated· AstraZeneca logo

AstraZeneca·Phase 2/3·NCT04999020·38 enrolled

Pneumonia1 trial

Efficacy and Safety Study of IV Ravulizumab in Patients With COVID-19 Severe Pneumonia

Terminated· AstraZeneca logo

AstraZeneca·Phase 3·NCT04369469·202 enrolled

Hypertensive1 trial

Ravulizumab in Pregnancies Complicated by Severe Hypertensive Disorders

Recruiting·MMayo Clinic·Phase 2·NCT06333652·14 enrolled

Neuromyelitis Optica1 trial

Study of the Clinical and Radiological Impact of Ravulizumab in People With Neuromyelitis Optica Spectrum Disorder

Recruiting·UUniversity of Texas Southwestern Medical Center··NCT06398158·35 enrolled

Lupus Nephritis1 trial

Study of Ravulizumab in Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN)

Terminated· AstraZeneca logo

AstraZeneca·Phase 2·NCT04564339·123 enrolled

Kidney Transplant1 trial

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

Recruiting· AstraZeneca logo

AstraZeneca·Phase 3·NCT06830798·450 enrolled

Adult1 trial

Pharmacokinetic Study of Ravulizumab Administered Subcutaneously With Recombinant Human Hyaluronidase PH20 (rHuPH20) in Healthy Adult Volunteers

Completed· AstraZeneca logo

AstraZeneca·Phase 1·NCT05396742·49 enrolled

Chronic Kidney Disease1 trial

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

Completed· AstraZeneca logo

AstraZeneca·Phase 3·NCT05746559·544 enrolled

NMOSD1 trial

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

Not yet recruiting·MMassachusetts General Hospital·Approved·NCT07010302·540 enrolled

Neuromyelitis Optica Spectrum Disorder1 trial

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)

Active, not recruiting· AstraZeneca logo

AstraZeneca·Preclinical·NCT05346354·12 enrolled

Generalized Myasthenia Gravis1 trial

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)

Not yet recruiting· AstraZeneca logo

AstraZeneca·Phase 3·NCT07596784·20 enrolled

Immunoglobulin A Nephropathy1 trial

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants With Immunoglobulin A Nephropathy (IgAN)

Active, not recruiting· AstraZeneca logo

AstraZeneca·Phase 3·NCT06291376·579 enrolled

Regulatory

FDA Approval Timeline[4]

Dec 21, 2018

ULTOMIRIS

BLA BLA761108

Sources

References

[1]
ClinicalTrials.gov — 43 trial records for Ravulizumab. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Ravulizumab compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Ravulizumab. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (BLA BLA761108). U.S. Food & Drug Administration.
[5]
SEC EDGAR — AstraZeneca company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Complement C5 Inhibitor[2]
Modality: Monoclonal antibody[2]
Highest Phase: Approved[1]
Company: AstraZeneca

Ultomiris

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2027

Q2 2027

Q3 2027

Trials by Indication[1]

Paroxysmal Nocturnal Hemoglobinuria12 trials

Myasthenia Gravis5 trials

Atypical Hemolytic-Uremic Syndrome3 trials

Thrombotic Microangiopathy2 trials

Pediatric2 trials

COVID-192 trials

Efficacy and Safety2 trials

Treatment Outcome1 trial

Anemia1 trial

ART1 trial

Dermatomyositis1 trial

Pneumonia1 trial

Hypertensive1 trial

Neuromyelitis Optica1 trial

Lupus Nephritis1 trial

Kidney Transplant1 trial

Adult1 trial

Chronic Kidney Disease1 trial

NMOSD1 trial

Neuromyelitis Optica Spectrum Disorder1 trial

Generalized Myasthenia Gravis1 trial

Immunoglobulin A Nephropathy1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

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