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.
Phosphorothioate oligonucleotide targeted to transthyretin (inotersen)
Treatment of ATTR amyloidosis
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Review of designation
|January 2023||The sponsor's address was updated.|
Documents related to this orphan designation evaluation
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: