Vamorolone clinical trials
Vamorolone is named as an intervention in 2 current clinical trials in our Ireland, UK, Spain, Netherlands, Belgium, Czechia, Israel and USA registers. 1 is recruiting patients now. The most common phase is Phase 4. The sponsor with the most trials is Santhera Pharmaceuticals (1). The most-studied condition is Duchenne Muscular Dystrophy.
Where Vamorolone is approved
All regulators →Of the three regulators tracked here, the first to approve Vamorolone was the US, on 26 Oct 2023. It is approved in 3 of the 3 regulators tracked here.
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.
Vamorolone patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Vamorolone, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- 7 listed patents; the last listed expires 16 Jul 2040 (US 11382922).
- Latest patent on the drug substance itself: 16 Jul 2040 (US 11382922).
- Exclusivity ODE-450: Orphan drug exclusivity: treatment of duchenne muscular dystrophy (dmd) in patients 2 years of age and older, until 26 Oct 2030.
- Exclusivity NCE: New chemical entity, until 26 Oct 2028.
- AGAMREE for treatment of Duchenne muscular dystrophy: until 15 Dec 2033. 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 Vamorolone
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 Vamorolone works
All drug targets →- Glucocorticoid receptor agonist AgonistTarget: NR3C1 nuclear receptor subfamily 3 group C member 1✓ In ChEMBL and the Guide to Pharmacology
- Mineralocorticoid receptor antagonist AntagonistTarget: NR3C2 nuclear receptor subfamily 3 group C member 2ChEMBL only
- Becker muscular dystrophyPhase 2
- Ulcerative colitisPhase 1/2
- Inflammatory bowel diseasePhase 1
- Multiple sclerosis symptom measurementPhase 1
- Rheumatoid arthritisPhase 1
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL2348780, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3.1), ligand 9247, 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 Vamorolone
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 1 technology appraisal of Vamorolone. 1 is current, and 1 of it recommends it for at least some patients.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Vamorolone for treating Duchenne muscular dystrophy in people 4 years and over NICE TA1031 | Recommended | 16 Jan 2025 | 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 Vamorolone
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- IEIreland: 1 product on the HPRA listEach product record links to the HPRA, which publishes the leaflet and SmPC.
- EUEuropean Union: AgamreeEMA product information: package leaflet and summary of product characteristics in every EU language.
- 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 Vamorolone
- ChEMBL
- CHEMBL2348780
- DrugBank
- DB15114
- PubChem CID
- 3035000
- CAS number
- 13209-41-1
- FDA UNII
- 8XP29XMB43
- Wikidata
- Q27089127
- Wikipedia
- Vamorolone
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 Vamorolone
2 trials| Study | Phase | Status | Lead sponsor | Registers |
|---|---|---|---|---|
|
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
NCT06564974 · Duchenne Muscular Dystrophy
|
Observational | Recruiting | Catalyst Pharmaceuticals, Inc | USA |
|
A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy
NCT06713135 · Duchenne Muscular Dystrophy
|
Phase 4 | Active, closed | Santhera Pharmaceuticals | Ireland, UK, Spain, Netherlands, Belgium, Czechia, Israel |
Registers 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.
Vamorolone 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)
1 single-substance product containing Vamorolone on the HPRA medicines list.
- AGAMREE 40 mg/ml oral suspensionSanthera Pharmaceuticals (Deutschland) GmbH · EU/1/23/1776/001
Supply shortages
No entry for Vamorolone in the EMA, FDA or German BfArM shortage lists we track.
Conditions studied with Vamorolone
1 conditionSponsors
lead sponsor per trial| Lead sponsor | Trials |
|---|---|
| Santhera Pharmaceuticals | 1 |
| Catalyst Pharmaceuticals, Inc | 1 |
By country register
A trial with sites in more than one country appears in each of those registers.
Related compounds
Questions about Vamorolone trials
How many clinical trials are testing Vamorolone?
2 current trials in our Ireland, UK, Spain, Netherlands, Belgium, Czechia, Israel and USA registers name Vamorolone as a drug or biological intervention, as of the registry sync on 10 November 2026. Completed and withdrawn studies are not counted.
Are Vamorolone trials recruiting patients?
Yes. 1 of the 2 trials is recruiting: 1 with sites in USA. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Who sponsors Vamorolone clinical trials?
Santhera Pharmaceuticals (1) and Catalyst Pharmaceuticals, Inc (1). The number in brackets is how many trials each organisation leads.
Are there Vamorolone 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 Vamorolone licensed in Ireland?
Yes. The HPRA medicines list includes 1 single-substance product containing Vamorolone, for example AGAMREE 40 mg/ml oral suspension (Santhera Pharmaceuticals (Deutschland) GmbH). A licence does not mean a product is currently marketed or reimbursed; see each product's record.
Is there a Vamorolone shortage?
Not in the lists we track: Vamorolone 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 Vamorolone 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 Vamorolone 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 Vamorolone (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.