EU/3/14/1250: Orphan designation for the treatment of ATTR amyloidosis
Phosphorothioate oligonucleotide targeted to transthyretin (inotersen)
Table of contents
Overview
On 26 March 2014, orphan designation (EU/3/14/1250) was granted by the European Commission to Isis USA Ltd, United Kingdom, for phosphorothioate oligonucleotide targeted to transthyretin for the treatment of ATTR amyloidosis.
In April 2016, Isis USA Ltd changed name to Ionis USA Ltd.
Phosphorothioate oligonucleotide targeted to transthyretin has been authorised in the EU as Tegsedi since 6 July 2018.
This medicine is now known as inotersen.
The sponsorship was transferred to Akcea Therapeutics UK Ltd., United Kingdom, in October 2018.
The sponsorship was transferred to Akcea Therapeutics Ireland Limited, Ireland, in February 2019.
Key facts
Active substance |
Phosphorothioate oligonucleotide targeted to transthyretin (inotersen)
|
Medicine name |
Tegsedi
|
Intended use |
Treatment of ATTR amyloidosis
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/14/1250
|
Date of designation |
26/03/2014
|
Sponsor |
Akcea Therapeutics Ireland Limited |
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Tegsedi at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.
More information is available in the
orphan medicine assessment report
.
Update history
Date | Update |
---|---|
January 2023 | The sponsor's address was updated. |
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: