EU/3/14/1249: Orphan designation for the treatment of familial chylomicronaemia syndrome

Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen)


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)
Intended use
Treatment of familial chylomicronaemia syndrome
Orphan designation status
EU designation number
Date of designation

Akcea Therapeutics Ireland Limited
St. James House
72 Adelaide Road
Dublin 2
Co. Dublin
D02 Y017

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 icon orphan medicine assessment report

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

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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