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 to bluebird bio (Germany) GmbH – Germany, in July 2018.

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 decision
29/04/2014
Outcome
Positive
Orphan decision 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 (Germany) GmbH
Josephspitalstraße 15
80331 Munich
Telephone: +49 174 263029
E-mail: patient.advocacy@bluebirdbio.com

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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