EU/3/14/1250: Orphan designation for the treatment of ATTR amyloidosis

Phosphorothioate oligonucleotide targeted to transthyretin (inotersen)

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
St. James House
72 Adelaide Road
Dublin 2
Co. Dublin
D02 Y017
Ireland
E-mail: info@akceatx.com

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 .

Update history

Date Update
January 2023 The sponsor's address was updated.

Documents related to this orphan designation evaluation

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:

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