On 2 February 2010, orphan designation (EU/3/09/720) was granted by the European Commission to Oxford BioMedica (UK) Ltd, United Kingdom, for lentiviral vector containing the human ABCA4 gene for the treatment of Stargardt's disease.
Lentiviral vector containing the human ABCA4 gene
Treatment of Stargardt's disease
|Orphan designation status||
|EU designation number||
|Date of designation||
|March 2023||The sponsorship was transferred from Sanofi-Aventis Groupe, France to Sanofi Winthrop Industrie, France.|
|June 2015||The sponsorship was transferred to Sanofi-Aventis Groupe, France.|
|June 2014||The sponsorship was transferred to Sanofi-Aventis Recherche & Développement, France.|
Documents related to this orphan designation evaluation
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: