EU/3/16/1665: Orphan designation for the treatment of retinitis pigmentosa caused by mutations in the RPGR gene
Recombinant adeno-associated viral vector containing the human RPGR gene
Table of contents
Overview
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.
Key facts
Active substance |
Recombinant adeno-associated viral vector containing the human RPGR gene
|
Intended use |
Treatment of retinitis pigmentosa caused by mutations in the RPGR gene
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/16/1665
|
Date of designation |
30/05/2016
|
Sponsor |
FGK Representative Service GmbH |
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Update history
Date | Update |
---|---|
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. |
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: