EU/3/16/1660

About

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.

Chimeric monoclonal antibody against GD2 has been authorised in the EU as Dinutuximab beta EUSA since 8 May 2017.

The sponsorship was transferred to EUSA Pharma (UK) Limited, United Kingdom, in September 2017.

The sponsorship was transferred to Orchard Therapeutics Ltd, United Kingdom in July 2018.

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

Key facts

Active substance
Autologous CD34+ cells transduced with lentiviral vector encoding the human beta globin gene
Disease / condition
Treatment of beta thalassaemia intermedia and major
Date of first decision
30/05/2016
Outcome
Positive
EU designation number
EU/3/16/1660

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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