EU/3/05/313

About

On 26 August 2005, orphan designation (EU/3/05/313) was granted by the European Commission to Fondazione Telethon, Italy, for autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene for the treatment of severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency.

The sponsorship was transferred to Glaxo Group Limited, United Kingdom, in June 2011 and subsequently to GlaxoSmithKline Trading Services Limited, Ireland, in July 2014.

Update: autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene has been authorised in the EU as Strimvelis since 26 May 2016.

More information on Strimvelis can be found in the European public assessment report (EPAR) on the Agency's website.

The sponsorship was transferred to Orchard Therapeutics (Netherlands) B.V. The Netherlands, in September 2018.

Key facts

Active substance
Autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene
Disease / condition
Treatment of severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency
Date of first decision
26/08/2005
Outcome
Positive
EU designation number
EU/3/05/313

Review of designation

On 8 April 2016, the Committee for Orphan Medicinal Products (COMP) completed its review of the designation EU/3/05/313 for Strimvelis (autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene) as an orphan medicinal product for the treatment of severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. The COMP recommended that the orphan designation of the medicine be maintained1.


1The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with the same therapeutic indication cannot be placed on the market.

Sponsor's contact details

Orchard Therapeutics (Netherlands) B.V.
Prins Bernhardplein 200
1097 JB Amsterdam
The Netherlands
Tel. +31 6570 41430
E-mail: info@orchard-tx.com

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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