EU/3/09/671: Orphan designation for the treatment of Cushing's disease



On 8 October 2009, orphan designation (EU/3/09/671) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for pasireotide for the treatment of Cushing's disease.

Pasireotide in treatment of Cushing's disease has been authorised in the EU as Signifor since 24 April 2012.

The sponsorship was transferred to Novartis Europharm Limited, Ireland, in May 2018.

The sponsorship was transferred to Recordati Rare Diseases, France in May 2020.

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in April 2022 at the end of the 10-year period of market exclusivity.

Key facts

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

Recordati Rare Diseases   
Immeuble Le Wilson
70 Avenue Du General De Gaulle
92800 Puteaux
Tel. +33 1 4773 9527

Review of designation

During its meeting of 7-8 February 2012, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/09/671 for Signifor (pasireotide) as an orphan medicinal product for the treatment of Cushing's disease. 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. As other satisfactory methods of treatment for patients with this condition are authorised in the European Union (EU), the COMP also looked at the significant benefit of the product over existing treatments. 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: Signifor (pasireotide) for the treatment of Cushing’s disease (PDF/116.58 KB)


    First published: 29/06/2012
    Last updated: 14/06/2022

  • 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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