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.
Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen)
Treatment of familial chylomicronaemia syndrome
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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.
More information is available in the orphan medicine assessment report .
For recommended INNs since designation please refer to the instructions in the PIS table above.
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: