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.

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 decision
26/10/2018
Outcome
Positive
Orphan decision 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

Fondazione Telethon
Via Varese 16b
00185 Roma
Italy
Tel. +39 06 44 01 51
E-mail: info@telethon.it

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;

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