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

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
Via Varese 16 B
00185 Rome
RM
Italy
E-mail: regulatory@telethon.it

 

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
September 2022The sponsorship was transferred from Orchard Therapeutics (Netherlands) B.V., the Netherlands to Fondazione Telethon Ets, Italy.
July 2022The sponsor's address was updated in July 2022.
April 2019The sponsorship was transferred to Orchard Therapeutics (Netherlands) B.V., the Netherlands.
July 2018The 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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