On 4 July 2014, orphan designation (EU/3/14/1283) was granted by the European Commission to Fondazione Telethon, Italy, for mixture of two adeno-associated viral vectors of serotye 8 containing the 5'-half sequence of human ABCA4 gene and the 3'-half sequence of human ABCA4 gene for the treatment of Stargardt's disease.
For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.
Mixture of two adeno-associated viral vectors of serotye 8 containing the 5'-half sequence of human ABCA4 gene and the 3'-half sequence of human ABCA4 gene
Treatment of Stargardt's disease
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
|November 2022||The sponsor's name was changed from Fondazione Telethon to Fondazione Telethon Ets in November 2022.|
Documents related to this orphan designation evaluation
EU/3/14/1283: Public summary of opinion on orphan designation: Mixture of two adeno-associated viral vectors of serotye 8 containing the 5’-half sequence of human ABCA4 gene and the 3’-half sequence of human ABCA4 gene f... (PDF/132.11 KB)
First published: 16/07/2014
Last updated: 26/03/2015
EMA/COMP/318161/2014 Rev. 1
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: