On 30 May 2016, orphan designation (EU/3/16/1665) was granted by the European Commission to TMC Pharma Services Ltd, United Kingdom, for recombinant adeno-associated viral vector containing the human RPGR gene for the treatment of retinitis pigmentosa caused by mutations in the RPGR gene.
Recombinant adeno-associated viral vector containing the human RPGR gene
Treatment of retinitis pigmentosa caused by mutations in the RPGR gene
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
|March 2023||The sponsorship was transferred from Propharma Group The Netherlands B.V. to FGK Representative Service GmbH.|
|October 2022||The sponsorship was transferred to Propharma Group The Netherlands B.V., Netherlands.|
|February 2020||The sponsorship was transferred to Diamond Pharma Services Ireland Limited, Ireland.|
|April 2019||The sponsorship was transferred to Diamond Roc EOOD, Bulgaria.|
Documents related to this orphan designation evaluation
EU/3/16/1665: Public summary of opinion on orphan designation: Recombinant adeno-associated viral vector containing the human RPGR gene for the treatment of retinitis pigmentosa caused by mutations in the RPGR gene (PDF/186.99 KB)
First published: 22/06/2016
Last updated: 22/06/2016
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: