Eplontersen clinical trials
Eplontersen is named as an intervention in 4 current clinical trials in our USA, France, Spain, Italy, Sweden, Canada, China, UK, Germany, Portugal, Brazil, Japan, Australia, Belgium, Denmark, Poland, Austria, Czechia, Israel, Turkey and Taiwan registers. None are recruiting at the moment. The most common phase is Phase 3. The sponsor with the most trials is Ionis Pharmaceuticals, Inc (2). The most-studied condition is Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM).
Where Eplontersen is approved
All regulators →Of the three regulators tracked here, the first to approve Eplontersen was the US, on 21 Dec 2023. It is approved in 3 of the 3 regulators tracked here.
- 1 EU orphan designation, most recently for treatment of transthyretin-mediated amyloidosis (13 Oct 2023, withdrawn)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.
Eplontersen patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Eplontersen, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- 3 listed patents; the last listed expires 25 Aug 2034 (US 10683499).
- Latest patent on the drug substance itself: 25 Aug 2034 (US 10683499).
- Exclusivity ODE-461: Orphan drug exclusivity: treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, until 21 Dec 2030.
- Exclusivity NCE: New chemical entity, until 21 Dec 2028.
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 Eplontersen
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 Eplontersen works
All drug targets →- Transthyretin mRNA antisense inhibitor Antisense inhibitorTarget: TTR transthyretinChEMBL only
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL4594334, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3.1), ligand 13606, 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 Eplontersen
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 1 technology appraisal of Eplontersen. 1 is current, and 1 of it recommends it for at least some patients.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Eplontersen for treating hereditary transthyretin-related amyloidosis NICE TA1020 | Recommended | 27 Nov 2024 | 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 Eplontersen
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: WainzuaEMA 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.
Trials by status
Trials by phase
Clinical trials of Eplontersen
4 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.
Eplontersen 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 Eplontersen on the HPRA medicines list.
- Wainzua 45 mg solution for injection in pre-filled penAstraZeneca AB · EU/1/24/1875/001
Supply shortages
No entry for Eplontersen in the EMA, FDA or German BfArM shortage lists we track.
Conditions studied with Eplontersen
4 conditionsSponsors
lead sponsor per trial| Lead sponsor | Trials |
|---|---|
| Ionis Pharmaceuticals, Inc | 2 |
| AstraZeneca | 2 |
By country register
A trial with sites in more than one country appears in each of those registers.
Related compounds
Questions about Eplontersen trials
How many clinical trials are testing Eplontersen?
4 current trials in our USA, France, Spain, Italy, Sweden, Canada, China, UK, Germany, Portugal, Brazil, Japan, Australia, Belgium, Denmark, Poland, Austria, Czechia, Israel, Turkey and Taiwan registers name Eplontersen (also registered as ION-682884) as a drug or biological intervention, as of the registry sync on 9 November 2026. Completed and withdrawn studies are not counted.
Are Eplontersen trials recruiting patients?
Not at the moment. None of the 4 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 Eplontersen clinical trials?
Ionis Pharmaceuticals, Inc (2) and AstraZeneca (2). The number in brackets is how many trials each organisation leads.
Is Eplontersen licensed in Ireland?
Yes. The HPRA medicines list includes 1 single-substance product containing Eplontersen, for example Wainzua 45 mg solution for injection in pre-filled pen (AstraZeneca AB). A licence does not mean a product is currently marketed or reimbursed; see each product's record.
Is there a Eplontersen shortage?
Not in the lists we track: Eplontersen 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 Eplontersen 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 Eplontersen 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 Eplontersen (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 9 Nov 2026. How every register is built: methodology · fixes we have made: corrections log.