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)

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
Via Varese 16 B
00185 Rome
RM
Italy
E-mail: regulatory@telethon.it

 

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
July 2022The sponsor's address was updated in July 2022.
March 2023The sponsorship was transferred from Orchard Therapeutics (Netherlands) B.V., Netherlands to Fondazione Telethon Ets, Italy

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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