On 12 March 2013, orphan designation (EU/3/13/1107) was granted by the European Commission to TMC Pharma Services Ltd, United Kingdom, for recombinant adeno-associated viral vector containing the human retinoschisin gene for the treatment of X-linked juvenile retinoschisis.
The sponsorship was transferred to Diamond Roc EOOD, Bulgaria, in April 2019.
The sponsorship was transferred to Diamond Pharma Services Ireland Limited in February 2020.
EU/3/13/1107: Public summary of opinion on orphan designation: Recombinant adeno-associated viral vector containing the human retinoschisin gene for the treatment of X-linked juvenile retinoschisis (PDF/126.79 KB)
First published: 05/04/2013
Last updated: 05/04/2013
Recombinant adeno-associated viral vector containing the human retinoschisin gene
|Disease / condition||
Treatment of X-linked juvenile retinoschisis
|Date of first decision||
|EU designation number||
Review of designation
Sponsor's contact details
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.