EU/3/02/127: Orphan designation for the treatment of inborn errors in primary bile acid synthesis

cholic acid

Overview

On 18 December 2002, orphan designation (EU/3/02/127) was granted by the European Commission to Agence Générale des Equipements et Produits de Santé - Etablissement Pharmaceutique des Hôpitaux de Paris (AGEPS - EPHP), France, for cholic acid for the treatment of inborn errors in primary-bile-acid synthesis.

The sponsorship was transferred to Laboratoires C.T.R.S., France, in July 2007.

Cholic acid has been authorised in the EU as Orphacol since 12 September 2013.

Key facts

Active substance
cholic acid
Medicine name
Orphacol
Intended use
Treatment of inborn errors in primary bile acid synthesis
Orphan designation status
Positive
EU designation number
EU/3/02/127
Date of designation
18/12/2002
Sponsor
Laboratoires C.T.R.S.
63 rue de l’Est
92100 Boulogne Billancourt
France
Tel. + 33 1412 20970
Fax + 33 1412 20236
E-mail: ctrs@ctrs.fr

Review of designation

During its meeting of 11-12 January 2011, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/02/127 for Orphacol (cholic acid) as an orphan medicinal product for the treatment of inborn errors in primary-bile-acid synthesis. 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 satisfactory methods of treatment. The COMP recommended that the orphan designation of the medicine be maintained*.


*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 European Union (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: Orphacol (cholic acid) for the treatment of inborn errors in primary-bile-acid synthesis (PDF/87.14 KB)

    Adopted

    First published: 10/10/2013
    Last updated: 10/10/2013
    EMA/COMP/10667/2011

  • 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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