EU/3/14/1249: Orphan designation for the treatment of familial chylomicronaemia syndrome
Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen)
Table of contents
Overview
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 |
Positive
|
EU designation number |
EU/3/14/1249
|
Date of designation |
19/02/2014
|
Sponsor |
Akcea Therapeutics Ireland Limited |
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
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. |
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: