Donidalorsen clinical trials
Donidalorsen is named as an intervention in 2 current clinical trials in our USA, Spain, Italy, Poland, UK, Germany, France, Netherlands, Belgium, Turkey, Canada, Israel and Australia registers. 1 is recruiting patients now. All registered phases are Phase 3. The lead sponsor is Ionis Pharmaceuticals, Inc. The most-studied condition is Hereditary Angioedema.
Where Donidalorsen is approved
All regulators →Of the three regulators tracked here, the first to approve Donidalorsen was the US, on 21 Aug 2025. It is approved in 3 of the 3 regulators tracked here.
- 1 EU orphan designation, most recently for treatment of hereditary angioedema (19 Feb 2024, withdrawn)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.
Donidalorsen patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Donidalorsen, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- 5 listed patents; the last listed expires 1 May 2035 (US 10294477).
- Latest patent on the drug substance itself: 1 May 2035 (US 10294477).
- Exclusivity ODE-550: Orphan drug exclusivity: prophylaxis to prevent attacks of hereditary angioedema (hae) in adult and pediatric patients 12 years of age and …, until 21 Aug 2032.
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.
How Donidalorsen works
All drug targets →- COVID-19Phase 2
- Acute respiratory distress syndromePhase 2
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL4650236, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3.1), ligand 13610, 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 Donidalorsen
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 1 technology appraisal of Donidalorsen. 1 is current, and 1 of it recommends it for at least some patients.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Donidalorsen for preventing recurrent attacks of hereditary angioedema in people 12 years and over NICE TA1183 | Optimised | 19 Aug 2026 | 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 Donidalorsen
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: DawnzeraEMA 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 Donidalorsen
2 trials| Study | Phase | Status | Lead sponsor | Registers |
|---|---|---|---|---|
|
Donidalorsen Treatment in Children With Hereditary Angioedema
NCT07298447 · Hereditary Angioedema (HAE)
|
Phase 3 | Recruiting | Ionis Pharmaceuticals, Inc | USA, Spain, Italy, Poland, Australia |
|
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
NCT05392114 · Hereditary Angioedema
|
Phase 3 | Active, closed | Ionis Pharmaceuticals, Inc | UK, USA, Germany, France, Spain, Italy, Netherlands, Belgium, Poland, Turkey, Canada, 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.
Donidalorsen 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 Donidalorsen on the HPRA medicines list.
- Dawnzera 80mg solution for injection in pre-filled penOtsuka Pharmaceutical Netherlands B.V. · EU/1/25/2001/001-002
Supply shortages
No entry for Donidalorsen in the EMA, FDA or German BfArM shortage lists we track.
Recent approvals
- DawnzeraEMA · 19 Jan 2026 · Otsuka Pharmaceutical Netherlands B.V.
Conditions studied with Donidalorsen
2 conditionsSponsors
lead sponsor per trial| Lead sponsor | Trials |
|---|---|
| Ionis Pharmaceuticals, Inc | 2 |
By country register
A trial with sites in more than one country appears in each of those registers.
Related compounds
Questions about Donidalorsen trials
How many clinical trials are testing Donidalorsen?
2 current trials in our USA, Spain, Italy, Poland, UK, Germany, France, Netherlands, Belgium, Turkey, Canada, Israel and Australia registers name Donidalorsen (also registered as ISIS 721744) as a drug or biological intervention, as of the registry sync on 10 November 2026. Completed and withdrawn studies are not counted.
Are Donidalorsen trials recruiting patients?
Yes. 1 of the 2 trials is recruiting: 1 with sites in USA, 1 with sites in Spain, 1 with sites in Italy, 1 with sites in Poland and 1 with sites in Australia. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Who sponsors Donidalorsen clinical trials?
The lead sponsor is Ionis Pharmaceuticals, Inc. The number in brackets is how many trials each organisation leads.
Is Donidalorsen licensed in Ireland?
Yes. The HPRA medicines list includes 1 single-substance product containing Donidalorsen, for example Dawnzera 80mg solution for injection in pre-filled pen (Otsuka Pharmaceutical Netherlands B.V.). A licence does not mean a product is currently marketed or reimbursed; see each product's record.
Is there a Donidalorsen shortage?
Not in the lists we track: Donidalorsen 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 Donidalorsen 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 Donidalorsen 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 Donidalorsen (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.