Eculizumab clinical trials
Eculizumab is named as an intervention in 12 current clinical trials in our China, Germany, France, UK, Spain, Netherlands, Poland, Portugal, Turkey, Brazil, Japan, South Korea, Taiwan, Singapore, Ireland, USA, Italy, Belgium, Sweden, Czechia and Mexico registers. 5 are recruiting patients now. The most common phase is Phase 3. The sponsor with the most trials is Hoffmann-La Roche (2). The most-studied condition is Paroxysmal Nocturnal Hemoglobinuria. The EU clinical trials register (CTIS) separately lists 1 authorised or ongoing trial.
Where Eculizumab is approved
All regulators →Of the three regulators tracked here, the first to approve Eculizumab was the US, on 16 Mar 2007. It is approved in 3 of the 3 regulators tracked here.
- 2 FDA-approved biosimilars, 2 interchangeable: Bkemv, Epysqli
- 8 EU orphan designations, most recently for treatment of Guillain-Barré syndrome (11 Oct 2022, withdrawn)
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
Eculizumab patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Eculizumab, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- Soliris for treatment of myasthenia gravis: until 17 Aug 2029 (includes 24 months' paediatric extension). Union Register
- Soliris for treatment of neuromyelitis optica spectrum disorders: until 28 Aug 2029. Union Register
Reproduced from the FDA Orange Book and Purple Book (checked 8 Nov 2026) and the European Commission's Union Register of medicinal products (CC BY 4.0; the EU expiry date is calculated exactly as the Commission's register displays it). Listed patents and dates are as submitted to or published by the regulator and may not reflect litigation, invalidation, settlements, lapsed fees or later extensions. This is not legal advice or a freedom-to-operate opinion, and it does not say when a generic or biosimilar will launch.
What health systems spend on Eculizumab
All medicines →From NHSBSA Prescription Cost Analysis (Open Government Licence v3.0) and CMS State Drug Utilization Data. Both are gross amounts: neither system publishes its discounts or rebates per drug. Products combining several substances are not counted here.
How Eculizumab works
All drug targets →- Complement C5 inhibitor InhibitorTarget: C5 complement C5ChEMBL only
- Guillain-Barre syndromePhase 3
- ThromboembolismPhase 3
- Acute kidney injuryPhase 3
- Hemolytic-uremic syndromePhase 3
- Malignant hypertensionPhase 3
- Myasthenia gravisPhase 3
- Optic neuritisPhase 3
- Thrombotic microangiopathyPhase 3
- COVID-19Phase 2
- ThrombocytopeniaPhase 2
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL1201828, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3.1), ligand 6884, CC BY-SA 4.0. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
NICE appraisals of Eculizumab
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 2 technology appraisals of Eculizumab. 2 are current.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Eculizumab for treating relapsing neuromyelitis optica (terminated appraisal) NICE TA647 | Terminated (no company submission) | 2 Sep 2020 | Current |
| Eculizumab for treating refractory myasthenia gravis (terminated appraisal) NICE TA636 | Terminated (no company submission) | 30 Jun 2020 | Current |
NICE decisions apply to the NHS in England. In Ireland the HSE decides on reimbursement, advised by the National Centre for Pharmacoeconomics. Titles, categories and dates link to the guidance on nice.org.uk. © NICE 2026 technology appraisal guidance. Available from www.nice.org.uk/guidance. All rights reserved. Subject to Notice of rights. NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.
Patient leaflets for Eculizumab
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- IEIreland: 3 products on the HPRA listEach product record links to the HPRA, which publishes the leaflet and SmPC.
- EU
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
Reference identifiers for Eculizumab
- ATC code
- L04AA25
- ChEMBL
- CHEMBL1201828
- DrugBank
- DB01257
- CAS number
- 219685-50-4
- FDA UNII
- A3ULP0F556
- Wikidata
- Q421617
- Wikipedia
- Eculizumab
Codes that identify this medicine in other databases, from Wikidata (CC0), matched by its ChEMBL ID. The ATC code is the WHO classification of what the medicine is used for.
Trials by status
Trials by phase
Clinical trials of Eculizumab
12 trialsRegisters list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Trial titles and statuses are copied from the registry as published.
Eculizumab in the EU trials register (CTIS)
1 trialAuthorised or ongoing trials in the EU Clinical Trials Information System that list a product containing Eculizumab. Many trials are registered in both CTIS and ClinicalTrials.gov, so these are shown separately rather than added to the count above. Trials with a site in Ireland are listed first.
| Study | Phase | Status | Sponsor | Countries |
|---|---|---|---|---|
|
Phase 1/2 Open-Label Trial to Evaluate the Safety, Tolerability, Biological Activity, and Clinical Efficacy of intracisterna Magna administration of an AAV9 Vector Encoding Human ASAH1 (GNT0009) in Patients with Spinal Muscular Atrophy with Progressive Myoclonic Epilepsy (SMA-PME)
2026-527323-10-00 · CTIS ↗ · Nervous System Diseases
|
Phase 1 | Authorised | Genethon | France |
Eculizumab across our data
Ireland · EU · USThe same active substance in our other registers, matched by its exact name. Each entry links to the register record it comes from.
Licensed in Ireland (HPRA)
3 single-substance products containing Eculizumab on the HPRA medicines list.
- BEKEMV 300 mg Concentrate for solution for infusionAmgen Technology (Ireland) Unlimited Company · EU/1/23/1727/001
- Epysqli 300 mg concentrate for solution for infusionSamsung Bioepis NL B.V. · EU/1/23/1735/001
- Soliris 300 mg concentrate for solution for infusionAlexion Europe SAS · EU/1/07/393/001
Supply shortages
No entry for Eculizumab in the EMA, FDA or German BfArM shortage lists we track.
Conditions studied with Eculizumab
8 conditionsSponsors
lead sponsor per trialBy country register
A trial with sites in more than one country appears in each of those registers. CTIS does not publish a recruiting flag; its trials may also appear in the registers above.
Related compounds
Questions about Eculizumab trials
How many clinical trials are testing Eculizumab?
12 current trials in our China, Germany, France, UK, Spain, Netherlands, Poland, Portugal, Turkey, Brazil, Japan, South Korea, Taiwan, Singapore, Ireland, USA, Italy, Belgium, Sweden, Czechia and Mexico registers name Eculizumab as a drug or biological intervention, as of the registry sync on 10 November 2026. Completed and withdrawn studies are not counted.
Are Eculizumab trials recruiting patients?
Yes. 5 of the 12 trials are recruiting: 3 with sites in China, 1 with sites in Germany and 1 with sites in France. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Who sponsors Eculizumab clinical trials?
Hoffmann-La Roche (2), Bisirna Therapeutics (Suzhou) Co., Ltd (1), Tianjin Medical University General Hospital (1) and Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd (1), among 11 lead sponsors in total. The number in brackets is how many trials each organisation leads.
Are there Eculizumab clinical trials in Ireland?
Yes. 1 trial in our Ireland register (ClinicalTrials.gov). Some may be the same study registered in both. See the Irish clinical trials register for sites and contacts.
Is Eculizumab licensed in Ireland?
Yes. The HPRA medicines list includes 3 single-substance products containing Eculizumab, for example BEKEMV 300 mg Concentrate for solution for infusion (Amgen Technology (Ireland) Unlimited Company). A licence does not mean a product is currently marketed or reimbursed; see each product's record.
Is there a Eculizumab shortage?
Not in the lists we track: Eculizumab has no entry in the EMA, FDA or German BfArM shortage registers as of their latest sync.
Where does this information come from?
Every trial, status, phase and sponsor on this page is copied from public ClinicalTrials.gov records by a daily sync. Nothing here describes how Eculizumab works or whether it is effective, and it is not medical advice.
How this page is built
Everything on this page is copied from public ClinicalTrials.gov registry records — it is not written from memory, and nothing here describes how Eculizumab works or whether it is effective. Each trial links to its own record so you can check it yourself.
- ClinicalTrials.gov (U.S. National Library of Medicine) ↗ Trial IDs, titles, intervention names and synonyms, phase, overall status, lead sponsor and conditions for every trial on this page
This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Eculizumab (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The ClinicalTrials.gov data on this page was last retrieved on 10 Nov 2026. How every register is built: methodology · fixes we have made: corrections log.