EU/3/12/998

About

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
Disease / condition
Treatment of Wiskott-Aldrich syndrome
Date of first decision
06/06/2012
Outcome
Positive
EU designation number
EU/3/12/998

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Orchard Therapeutics (Netherlands) B.V.
Prins Bernhardplein 200
1097 JB Amsterdam
The Netherlands
Tel. +31 6570 41430
E-mail: info@orchard-tx.com

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;

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