EU/3/05/313: Orphan designation for the treatment of severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency

Autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene

Overview

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.

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.

Key facts

Active substance
Autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene
Intended use
Treatment of severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency
Orphan designation status
Positive
EU designation number
EU/3/05/313
Date of designation
26/08/2005
Sponsor

Fondazione Telethon Ets
Via Varese 16 B
00185 Rome
RM
Italy
E-mail: regulatory@telethon.it

 

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.

Update history

DATEUpdate
June 2011The sponsorship was transferred to Glaxo Group Limited, United Kingdom, in June 2011.
July 2014The sponsorship was transferred to GlaxoSmithKline Trading Services Limited, Ireland, in July 2014.
September 2018The sponsorship was transferred to Orchard Therapeutics (Netherlands) B.V., The Netherlands, in September 2018.
July 2022The sponsor's address was updated in July 2022.
June 2023The sponsorship was transferred to Fondazione Telethon Ets, Italy, in June 2023.

Documents related to this orphan designation evaluation

  • List item

    Recommendation for maintenance of orphan designation at the time of marketing authorisation: Strimvelis (autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene) for the treatment of... (PDF/79.29 KB)

    Adopted

    First published: 08/06/2016
    Last updated: 08/06/2016
    EMA/COMP/260673/2016

  • 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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