EU/3/13/1134

About

On 7 June 2013, orphan designation (EU/3/13/1134) was granted by the European Commission to Prof. Bobby Gaspar, United Kingdom, for autologous CD34+ cells transduced with a lentiviral vector containing the human ADA gene for the treatment of adenosine-deaminase-deficient severe combined immunodeficiency.

The sponsorship was transferred to Orchard Therapeutics Ltd, United Kingdom, in November 2017.

The sponsorship was transferred to Orchard Therapeutics (Netherlands) B.V., Netherlands, in March 2019.

Key facts

Active substance
Autologous CD34+ cells transduced with a lentiviral vector containing the human ADA gene
Disease / condition
Treatment of adenosine-deaminase-deficient severe combined immunodeficiency
Date of decision
07/06/2013
Outcome
Positive
Orphan decision number
EU/3/13/1134

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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