EU/3/09/720: Orphan designation for the treatment of Stargardt's disease

Lentiviral vector containing the human ABCA4 gene

Table of contents


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.

Key facts

Active substance
Lentiviral vector containing the human ABCA4 gene
Intended use
Treatment of Stargardt's disease
Orphan designation status
EU designation number
Date of designation

Sanofi Winthrop Industrie
82 avenue Raspail
94250 Gentilly

Update history

March 2023The sponsorship was transferred from Sanofi-Aventis Groupe, France to Sanofi Winthrop Industrie, France.
June 2015The sponsorship was transferred to Sanofi-Aventis Groupe, France.
June 2014The sponsorship was transferred to Sanofi-Aventis Recherche & Développement, France.

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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