EU/3/05/278: Orphan designation for the treatment of Duchenne muscular dystrophy

3-[5-(2-Fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid (ataluren)


This medicine is now known as ataluren.

On 27 May 2005, orphan designation (EU/3/05/278) was granted by the European Commission to The Matthews Consultancy Ltd, United Kingdom, for 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid for the treatment of Duchenne muscular dystrophy.

The sponsorship was transferred to Voisin Consulting S.A.R.L., France, in May 2007 and subsequently to PTC Therapeutics Limited, United Kingdom in April 2012. In October 2014 the sponsorship was transferred to PTC Therapeutics International Limited, Ireland.

3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid for the treatment of Duchenne muscular dystrophy has been authorised in the EU as Translarna since 31 July 2014.

Key facts

Active substance
3-[5-(2-Fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid (ataluren)
Medicine name
Intended use
Treatment of Duchenne muscular dystrophy
Orphan designation status
EU designation number
Date of designation

PTC Therapeutics International Limited
5th floor, 3 Grand Canal Plaza
Grand Canal Street Upper
Dublin 4, D04 EE70
Tel. +353 1 906 87 37

Review of designation

During its meeting of 10 to 12 June 2014, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/05/278 for Translarna (ataluren1) as an orphan medicinal product for the treatment of Duchenne muscular dystrophy. 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 maintained2.

1 Previously known as 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid.
2 The 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 a comparable therapeutic indication cannot be placed on the market.

Documents related to this orphan designation evaluation

  • List item

    Recommendation for maintenance of orphan designation at the time of marketing authorisation: Translarna (ataluren) for the treatment of Duchenne muscular dystrophy (PDF/78.35 KB)


    First published: 04/09/2014
    Last updated: 04/09/2014

  • 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.

    • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

    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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