EU/3/07/446: Orphan designation for the treatment of metachromatic leukodystrophy

Autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA

Overview

On 13 April 2007, orphan designation (EU/3/07/446) was granted by the European Commission to Fondazione Telethon, Italy, for autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA for the treatment of metachromatic leukodystrophy.

The sponsorship was transferred to GlaxoSmithKline Trading Services Limited, Ireland, in July 2014, to Orchard Therapeutics Ltd, United Kingdom in July 2018 and to Orchard Therapeutics (Netherlands) B.V., The Netherlands in March 2019.

Autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA has been authorised in the EU as Libmeldy since 17 December 2020.

Key facts

Active substance
Autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA
Intended use
treatment of metachromatic leukodystrophy
Orphan designation status
Positive
EU designation number
EU/3/07/446
Date of designation
13/04/2007
Sponsor

Orchard Therapeutics (Netherlands) B.V.
Prins Bernhardplein 200
1097 JB Amsterdam
The Netherlands
E-mail: regulatory@orchard-tx.com

Review of designation

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

More information is available in the PDF icon 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:

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