EU/3/13/1134: Orphan designation for the treatment of adenosine-deaminase-deficient severe combined immunodeficiency

Autologous CD34+ cells transduced with a lentiviral vector containing the human ADA gene

Overview

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.

 

Key facts

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

AdRes EU B.V.
Pietersbergweg 283
1105 BM Amsterdam
Noord-Holland
Netherlands
Tel: + 31207920069
E-mail: Office@adreseu.nl

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
March 2023The sponsorship was transferred from Orchard Therapeutics (Netherlands) B.V. to AdRes EU B.V., Netherlands.
July 2022The sponsor's address was updated in July 2022.
March 2019The sponsorship was transferred to Orchard Therapeutics (Netherlands) B.V., Netherlands.
November 2017The sponsorship was transferred to Orchard Therapeutics Ltd, United Kingdom.

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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