EU/3/14/1263

About

On 29 April 2014, orphan designation (EU/3/14/1263) was granted by the European Commission to bluebird bio France SARL, France, for autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human beta A-T87Q-globin gene for the treatment of sickle cell disease.

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

Key facts

Active substance
Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human beta A-T87Q-globin gene for the treatment of sickle cell disease
Disease / condition
The treatment of sickle cell disease
Date of first decision
29/04/2014
Outcome
Positive
EU designation number
EU/3/14/1263

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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