EU/3/12/965: Orphan designation for the treatment of Cushing's syndrome



On 23 April 2012, orphan designation (EU/3/12/965) was granted by the European Commission to Laboratoire HRA Pharma, France, for ketoconazole for the treatment of Cushing's syndrome.

Ketoconazole has been authorised in the EU as Ketoconazole HRA since 19 November 2014.

The sponsorship was transferred to HRA Pharma Rare Diseases, France in December 2019.

Key facts

Active substance
Medicine name
Ketoconazole HRA
Intended use
Treatment of Cushing's syndrome
Orphan designation status
EU designation number
Date of designation

HRA Pharma Rare Diseases
200 avenue de Paris
92320 Chatillon
Tel: +33 1 40 33 24 85

Review of designation

During its meeting of 7 to 9 October 2014, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/12/965 for Ketoconazole HRA (ketoconazole) as an orphan medicinal product for the treatment of Cushing's syndrome. 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. As other methods of treatment are authorised in the European Union (EU), the COMP also considered whether the medicine is of significant benefit to patients with Cushing's syndrome. 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 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: Ketoconazole HRA (ketoconazole) for the treatment of Cushing’s syndrome (PDF/78.88 KB)


    First published: 30/01/2015
    Last updated: 30/01/2015

  • 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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