Viltolarsen clinical trials
Viltolarsen is named as an intervention in 2 current clinical trials in our UK, China, Czechia, Norway, Mexico, Australia, USA and Canada registers. None are recruiting at the moment. The most common phase is Phase 3. The lead sponsor is NS Pharma, Inc. The most-studied condition is Duchenne Muscular Dystrophy.
Where Viltolarsen is approved
All regulators →Of the three regulators tracked here, the first to approve Viltolarsen was the US, on 12 Aug 2020.
- 1 EU orphan designation, most recently for treatment of Duchenne muscular dystrophy (4 Jun 2020, positive)
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.
Viltolarsen patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Viltolarsen, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- 2 listed patents; the last listed expires 3 Apr 2034 (US 9079934).
- Latest patent on the drug substance itself: 3 Apr 2034 (US 9079934).
- Exclusivity ODE-280: Orphan drug exclusivity: indicated for the treatment of duchenne muscular dystrophy (dmd) in patients who have a confirmed mutation of the …, until 12 Aug 2027.
Reproduced from the FDA Orange Book and Purple Book (checked 19 Oct 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 Viltolarsen
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.
Patient leaflets for Viltolarsen
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- 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.
Trials by status
Trials by phase
Clinical trials of Viltolarsen
2 trials| Study | Phase | Status | Lead sponsor | Registers |
|---|---|---|---|---|
|
Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
NCT04687020 · Duchenne Muscular Dystrophy
|
Phase 4 | Active, closed | NS Pharma, Inc | USA, Canada |
|
Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)
NCT04768062 · Duchenne Muscular Dystrophy
|
Phase 3 | Active, closed | NS Pharma, Inc | UK, China, Czechia, Norway, Mexico, Australia |
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.
Conditions studied with Viltolarsen
1 conditionSponsors
lead sponsor per trial| Lead sponsor | Trials |
|---|---|
| NS Pharma, Inc | 2 |
By country register
A trial with sites in more than one country appears in each of those registers.
Related compounds
Questions about Viltolarsen trials
How many clinical trials are testing Viltolarsen?
2 current trials in our UK, China, Czechia, Norway, Mexico, Australia, USA and Canada registers name Viltolarsen (also registered as NS-065/NCNP-01) as a drug or biological intervention, as of the registry sync on 21 October 2026. Completed and withdrawn studies are not counted.
Are Viltolarsen trials recruiting patients?
Not at the moment. None of the 2 trials in our registers is recruiting; they are active but closed to enrolment, not yet open, or enrolling by invitation. Only the study team can confirm whether a patient is eligible.
Who sponsors Viltolarsen clinical trials?
The lead sponsor is NS Pharma, Inc. The number in brackets is how many trials each organisation leads.
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 Viltolarsen 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 Viltolarsen 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 Viltolarsen (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 21 Oct 2026. How every register is built: methodology · fixes we have made: corrections log.