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