EU/3/18/2073

About

On 26 October 2018, orphan designation (EU/3/18/2073) was granted by the European Commission to Fondazione Telethon, Italy, for autologous CD34+ haematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the alpha-L-iduronidase cDNA for the treatment of mucopolysaccharidosis type I.

The sponsorship was transferred to Orchard Therapeutics (Netherlands) B.V., Netherlands, in January 2020.

Key facts

Active substance
autologous CD34+ haematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the alpha-L-iduronidase cDNA
Disease / condition
Treatment of mucopolysaccharidosis type I
Date of first decision
26/10/2018
Outcome
Positive
EU designation number
EU/3/18/2073

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

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

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