EU/3/12/1091

About

On 24 January 2013, orphan designation (EU/3/12/1091) was granted by the European Commission to bluebird bio France, France, for autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene for treatment of beta thalassaemia intermedia and major.

The sponsorship was transferred to bluebird bio (Germany) GmbH, Germany, in July 2018 and subsequently to bluebird bio (Netherlands) B.V., Netherlands, in February 2019.

Key facts

Active substance
Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene
Disease / condition
Treatment of beta thalassaemia intermedia and major
Date of decision
24/01/2013
Outcome
Positive
Orphan decision number
EU/3/12/1091

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

bluebird bio (Netherlands) B.V.
Stadsplateau 7
Utrecht
3521 AZ
Netherlands
Tel. +31 30 800 92 00
E-mail: patient.advocacy@bluebirdbio.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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