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

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
Heimeranstrasse 35
Schwanthalerhoehe-Laim 8
0339 Munich
Bavaria
Germany
Email: tracanelli@agtc.com

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

DateUpdate
March 2023The sponsorship was transferred from Propharma Group The Netherlands B.V. to FGK Representative Service GmbH.
October 2022The sponsorship was transferred to Propharma Group The Netherlands B.V., Netherlands.
February 2020The sponsorship was transferred to Diamond Pharma Services Ireland Limited, Ireland.
April 2019The sponsorship was transferred to Diamond Roc EOOD, Bulgaria.

Documents related to this orphan designation evaluation

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:

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