EU/3/12/1003: Orphan designation for the treatment of adrenoleukodystrophy

Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA (elivaldogene autotemcel)

Overview

Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA was authorised in the EU as Skysona since 16 July 2021.

The marketing authorisation was withdrawn on 19 November 2021.

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products by the European Commission in November 2021 at the time of withdrawal of the marketing authorisation.

This medicine is now known as elivaldogene autotemcel.

On 6 June 2012, orphan designation (EU/3/12/1003) was granted by the European Commission to bluebird bio France, France, for autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA for the treatment of adrenoleukodystrophy.

The sponsorship was transferred to bluebird bio (Germany) GmbH – Germany, in July 2018.

The sponsorship was transferred to bluebird bio (Netherlands) B.V., the Netherlands, in February 2019.

Key facts

Active substance
Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA (elivaldogene autotemcel)
Intended use
Treatment of adrenoleukodystrophy
Orphan designation status
Withdrawn
EU designation number
EU/3/12/1003
Date of designation
06/06/2012
Sponsor

bluebird bio (Netherlands) B.V.
Stadsplateau 7
3521 AZ Utrecht
The Netherlands
Tel. +31 3080 09200
E-mail: patient.advocacy@bluebirdbio.com

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Skysona 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.

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