Vutrisiran clinical trials
Vutrisiran is named as an intervention in 3 current clinical trials in our UK, USA, France, Spain, Sweden, Portugal, South Korea, Ireland, Germany, Netherlands, Belgium, Denmark, Austria, Czechia, Norway, Canada, Japan, Australia, Italy, Brazil, Taiwan, Poland, Israel, Switzerland, Turkey and Mexico registers. 1 is recruiting patients now. All registered phases are Phase 3. The lead sponsor is Alnylam Pharmaceuticals. The most-studied condition is Transthyretin Amyloidosis (ATTR) With Cardiomyopathy.
Where Vutrisiran is approved
All regulators →Of the three regulators tracked here, the first to approve Vutrisiran was the US, on 13 Jun 2022. It is approved in 3 of the 3 regulators tracked here.
- 1 EU orphan designation, most recently for treatment of Stargardt's disease (10 Aug 2022, withdrawn)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.
Vutrisiran patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Vutrisiran, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- 13 listed patents; the last listed expires 28 Jul 2036 (US 11286486).
- Latest patent on the drug substance itself: 28 Jul 2036 (US 11286486).
- Exclusivity ODE-527: Orphan drug exclusivity: treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (attr-cm) in adults …, until 20 Mar 2032.
- Exclusivity ODE-212: Orphan drug exclusivity: indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, until 13 Jun 2029.
- Exclusivity I-964: New indication: treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovas…, until 20 Mar 2028.
- Exclusivity NCE: New chemical entity, until 13 Jun 2027.
- Amvuttra for treatment of transthyretin-mediated amyloidosis: until 16 Sep 2032. 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 Vutrisiran
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 Vutrisiran works
All drug targets →- Transthyretin mRNA rnai inhibitor Rnai inhibitorTarget: TTR transthyretinChEMBL only
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL4594511, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3.1), ligand 13616, 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 Vutrisiran
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 2 technology appraisals of Vutrisiran. 2 are current, and 2 of these recommend it for at least some patients.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Vutrisiran for treating transthyretin amyloidosis with cardiomyopathy NICE TA1115 | Recommended | 10 Dec 2025 | Current |
| Vutrisiran for treating hereditary transthyretin-related amyloidosis NICE TA868 | Recommended | 15 Feb 2023 | 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 Vutrisiran
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: AmvuttraEMA 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 Vutrisiran
3 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.
Vutrisiran 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 Vutrisiran on the HPRA medicines list.
- Amvuttra 25 mg solution for injection in pre-filled syringeAlnylam Netherlands B.V. · EU/1/22/1681/001
Supply shortages
No entry for Vutrisiran in the EMA, FDA or German BfArM shortage lists we track.
Conditions studied with Vutrisiran
3 conditionsSponsors
lead sponsor per trial| Lead sponsor | Trials |
|---|---|
| Alnylam Pharmaceuticals | 3 |
By country register
A trial with sites in more than one country appears in each of those registers.
Related compounds
Questions about Vutrisiran trials
How many clinical trials are testing Vutrisiran?
3 current trials in our UK, USA, France, Spain, Sweden, Portugal, South Korea, Ireland, Germany, Netherlands, Belgium, Denmark, Austria, Czechia, Norway, Canada, Japan, Australia, Italy, Brazil, Taiwan, Poland, Israel, Switzerland, Turkey and Mexico registers name Vutrisiran (also registered as ALN-TTRSC02) as a drug or biological intervention, as of the registry sync on 10 November 2026. Completed and withdrawn studies are not counted.
Are Vutrisiran trials recruiting patients?
Yes. 1 of the 3 trials is recruiting: 1 with sites in UK, 1 with sites in USA, 1 with sites in France, 1 with sites in Spain, 1 with sites in Sweden, 1 with sites in Portugal, 1 with sites in South Korea, 1 with sites in Ireland, 1 with sites in Germany, 1 with sites in Canada, 1 with sites in Japan, 1 with sites in Australia, 1 with sites in Italy, 1 with sites in Brazil, 1 with sites in Taiwan, 1 with sites in Switzerland, 1 with sites in Turkey and 1 with sites in Mexico. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Who sponsors Vutrisiran clinical trials?
The lead sponsor is Alnylam Pharmaceuticals. The number in brackets is how many trials each organisation leads.
Are there Vutrisiran clinical trials in Ireland?
Yes. 2 trials 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 Vutrisiran licensed in Ireland?
Yes. The HPRA medicines list includes 1 single-substance product containing Vutrisiran, for example Amvuttra 25 mg solution for injection in pre-filled syringe (Alnylam Netherlands B.V.). A licence does not mean a product is currently marketed or reimbursed; see each product's record.
Is there a Vutrisiran shortage?
Not in the lists we track: Vutrisiran 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 Vutrisiran 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 Vutrisiran 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 Vutrisiran (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.