Eculizumab

Generic Name: Eculizumab

Brand Names: Soliris, Epysqli, Bekemv

Drug Type: Biotech

CAS Number: 219685-50-4

Unique Ingredient Identifier: A3ULP0F556

Background: Eculizumab is a monoclonal antibody that targets complement protein C5. Binding to this protein prevents the activation of a complement terminal complex, which is used to treat a number of autoimmune conditions.

Eculizumab was granted FDA approval on 16 March 2007. In Q1 2023, the EMA's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion of two formulations of eculizumab indicated for the treatment of paroxysmal nocturnal hemoglobinuria.

Indication: Eculizumab is indicated in the US to treat paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis, atypical hemolytic uremic syndrome to inhibit complement-mediated thrombotic microangiopathy, and neuromyelitis optica spectrum disorder (NMOSD). It is also indicated in EU to treat PNH in both adult and pediatric patients.

Associated Conditions: Generalized Myasthenia Gravis, Neuromyelitis Optica Spectrum Disorders, Paroxysmal Nocturnal Haemoglobinuria (PNH), Thrombotic Microangiopathies

Clinical Trials

Comparative PK, Safety, Tolerability, Immunogenicity, and PD Profile Study of TUR03 and Soliris in Healthy Participants

Phase 1

Recruiting

Conditions: Healthy

Interventions: Drug: Soliris 300 MG in 30 ML Injection
Drug: TUR03 300 MG in 30 ML Injection

First Posted Date: 2023-05-18

Last Posted Date: 2023-11-24

Lead Sponsor: Turgut Ardika PTY LTD

Target Recruit Count: 120

Registration Number: NCT05863442

Locations: 🇦🇺
Q-Pharm Pty Limited, Herston, Queensland, Australia

Eculizumab in Hypertensive Emergency-associated Hemolytic Uremic Syndrome

Phase 3

Recruiting

Conditions: Hypertensive Emergency-associated Hemolytic Uremic Syndrome

Interventions: Drug: Soliris®
Drug: Renin angiotensin system blockers

First Posted Date: 2023-02-14

Last Posted Date: 2024-01-05

Lead Sponsor: Assistance Publique - Hôpitaux de Paris

Target Recruit Count: 66

Registration Number: NCT05726916

Locations: 🇫🇷
Tenon Hospital, Paris, France

Multicenter, Uncontrolled Pilot Study Evaluating the Efficacy of Eculizumab in the Treatment of Gemcitabine-induced Thrombotic Microangiopathies

Phase 3

Not yet recruiting

Conditions: Thrombotic Microangiopathies

Interventions: Drug: Eculizumab administration

First Posted Date: 2023-01-27

Last Posted Date: 2023-01-27

Lead Sponsor: University Hospital, Rouen

Target Recruit Count: 10

Registration Number: NCT05702996

Locations: 🇫🇷
Rouen University Hospital, Rouen, France
🇫🇷
Amiens University Hospital, Amiens, France

Study of NM8074 in Adult PNH Patients with Inadequate Response to Soliris

Phase 2

Not yet recruiting

Conditions: Paroxysmal Nocturnal Hemoglobinuria

Interventions: Drug: NM8074
Drug: Soliris

First Posted Date: 2022-12-12

Last Posted Date: 2025-03-11

Lead Sponsor: NovelMed Therapeutics

Target Recruit Count: 12

Registration Number: NCT05646563

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

Phase 3

Recruiting

Conditions: Paroxysmal Nocturnal Hemoglobinuria

Interventions: Drug: Ravulizumab
Drug: Pozelimab
Drug: Cemdisiran
Drug: Eculizumab

First Posted Date: 2021-11-24

Last Posted Date: 2025-05-01

Lead Sponsor: Regeneron Pharmaceuticals

Target Recruit Count: 190

Registration Number: NCT05133531

Locations: 🇨🇦
Toronto General Hospital, Toronto, Ontario, Canada
🇮🇳
Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus, New Delhi, Delhi, India
🇮🇳
Postgraduate Institute of Medical Education & Research (PGIMER), Chandigarh, Punjab, India

and more 60 locations

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

Phase 3

Terminated

Conditions: Paroxysmal Nocturnal Hemoglobinuria

Interventions: Drug: Cemdisiran
Drug: Eculizumab
Drug: Ravulizumab
Drug: Pozelimab

First Posted Date: 2021-11-23

Last Posted Date: 2025-04-08

Lead Sponsor: Regeneron Pharmaceuticals

Target Recruit Count: 2

Registration Number: NCT05131204

Locations: 🇺🇸
Regeneron Research Facility, Whittier, California, United States

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

Phase 2

Terminated

Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions: Drug: BCX9930
Drug: Eculizumab
Drug: Ravulizumab

First Posted Date: 2021-11-11

Last Posted Date: 2025-03-07

Lead Sponsor: BioCryst Pharmaceuticals

Target Recruit Count: 12

Registration Number: NCT05116774

Locations: 🇬🇧
Investigative Site, London, United Kingdom

Renal Function in Highly Sensitized Patients 1 Year After Desensitization With Imlifidase Prior to DD Kidney Tx

Phase 3

Active, not recruiting

Conditions: Kidney Transplantation in Highly Sensitized Patients

Interventions: Procedure: PLEX
Drug: Imlifidase
Drug: IVIg
Drug: Anti-CD20 antibodies
Drug: Eculizumab
Other: Remain on wait list

First Posted Date: 2021-06-22

Last Posted Date: 2024-11-21

Lead Sponsor: Hansa Biopharma AB

Target Recruit Count: 64

Registration Number: NCT04935177

Locations: 🇺🇸
Washington University School of Medicine, Saint Louis, Missouri, United States
🇺🇸
Georgetown Transplant Institute, Washington, District of Columbia, United States
🇺🇸
Columbia University, New York, New York, United States

and more 22 locations

Complement Regulation to Undo Systemic Harm in Preeclampsia

Phase 2

Terminated

Conditions: Preeclampsia
AHUS
PNH
Severe Preeclampsia
Pregnancy Related
Complement Abnormality
Eculizumab
HELLP Syndrome Third Trimester
HELLP
HELLP Syndrome