EU/3/16/1660: Orphan designation for the treatment of beta thalassaemia intermedia and major
Autologous CD34+ cells transduced with lentiviral vector encoding the human beta globin gene
Table of contents
Overview
On 30 May 2016, orphan designation (EU/3/16/1660) was granted by the European Commission to Fondazione Telethon, Italy, for autologous CD34+ cells transduced with lentiviral vector encoding the human beta globin gene for the treatment of beta thalassaemia intermedia and major.
Key facts
Active substance |
Autologous CD34+ cells transduced with lentiviral vector encoding the human beta globin gene
|
Intended use |
Treatment of beta thalassaemia intermedia and major
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/16/1660
|
Date of designation |
30/05/2016
|
Sponsor |
Fondazione Telethon Ets
|
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 |
---|---|
September 2022 | The sponsorship was transferred from Orchard Therapeutics (Netherlands) B.V., the Netherlands to Fondazione Telethon Ets, Italy. |
July 2022 | The sponsor's address was updated in July 2022. |
April 2019 | The sponsorship was transferred to Orchard Therapeutics (Netherlands) B.V., the Netherlands. |
July 2018 | 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: