EU/3/12/998: Orphan designation for the treatment of Wiskott-Aldrich syndrome
Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene (etuvetidigene autotemcel)
Table of contents
Overview
On 6 June 2012, orphan designation (EU/3/12/998) was granted by the European Commission to Fondazione Telethon, Italy, for autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene for the treatment of Wiskott-Aldrich syndrome.
The sponsorship was transferred to GlaxoSmithKline Trading Services Limited, Ireland, in December 2014 and subsequently to Orchard Therapeutics Ltd, in July 2018.
The sponsorship was transferred to Orchard Therapeutics (Netherlands) B.V., the Netherlands, in April 2019.
Key facts
Active substance |
Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene (etuvetidigene autotemcel)
|
Intended use |
Treatment of Wiskott-Aldrich syndrome
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/12/998
|
Date of designation |
06/06/2012
|
Sponsor |
Fondazione Telethon Ets
|
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 |
---|---|
July 2022 | The sponsor's address was updated in July 2022. |
March 2023 | The sponsorship was transferred from Orchard Therapeutics (Netherlands) B.V., Netherlands to Fondazione Telethon Ets, Italy |
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: