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
Disease / condition
Treatment of ATTR amyloidosis
Date of first decision
EU designation number

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 PDF icon orphan medicine assessment report .

Sponsor's contact details

Akcea Therapeutics Ireland Limited
Regus House
Harcourt Centre
Harcourt Road
Dublin 2
Tel. + 353 1477 3197

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.

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