EU/3/14/1249

About

On 19 February 2014, orphan designation (EU/3/14/1249) was granted by the European Commission to Isis USA Ltd, United Kingdom, for phosphorothioate oligonucleotide targeted to apolipoprotein C-III for treatment of familial chylomicronaemia syndrome.

In April 2016, Isis USA Ltd changed name to Ionis USA Ltd.

The sponsorship was transferred to Akcea Therapeutics UK Ltd., United Kingdom, in July 2017 and subsequently to Akcea Therapeutics Ireland Limited, Ireland, in March 2019.

 

Key facts

Active substance
Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen)
Disease / condition
Treatment of familial chylomicronaemia syndrome
Date of first decision
19/02/2014
Outcome
Positive
EU designation number
EU/3/14/1249

Review of designation

This medicine is now known as volanesorsen.

Phosphorothioate oligonucleotide targeted to apolipoprotein C-III for treatment of familial chylomicronemia syndrome has been authorised in the EU as Waylivra since 3 May 2019.

The Committee for Orphan Medicinal Products reviewed the orphan designation of Waylivra at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the PDF iconorphan medicine assessment report

For recommended INNs since designation please refer to the instructions in the PIS table above. 

Sponsor's contact details

Akcea Therapeutics Ireland Limited
Regus House
Harcourt Centre
Harcourt Road
Dublin 2
Ireland
Tel: + 353 1 477 3197
E-mail: JMacPherson@akceatx.com

 

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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