EU/3/12/1091: Orphan designation for the treatment of beta thalassaemia intermedia and major

Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene (betibeglogene autotemcel)

Overview

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.

Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene was authorised in the EU as Zynteglo on 29 May 2019.

The marketing authorisation was withdrawn on 29 March 2022.

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products by the European Commission in March 2022 at the time of withdrawal of the marketing authorisation.

Key facts

Active substance
Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene (betibeglogene autotemcel)
Intended use
Treatment of beta thalassaemia intermedia and major
Orphan designation status
Withdrawn
EU designation number
EU/3/12/1091
Date of designation
24/01/2013
Sponsor

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

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

The Committee for Orphan Medicinal Products reviewed the orphan designation of Zynteglo at the time of marketing authorisation and confirmed that the orphan designation should be maintained.

More information is available in the orphan medicine assessment report PDF icon Zynteglo - Orphan maintenance assessment report

Update history

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

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:

How useful was this page?

Add your rating