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.
Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene (etuvetidigene autotemcel)
Treatment of Wiskott-Aldrich syndrome
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
|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|
Documents related to this orphan designation evaluation
EU/3/12/998: Public summary of opinion on orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene for the treatment of Wiskott-Aldrich syndrome (PDF/128.66 KB)
First published: 04/07/2012
Last updated: 11/03/2015
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: