EU/3/09/653: Orphan designation for the treatment of atypical haemolytic uraemic syndrome (aHUS)

Eculizumab

Overview

On 24 July 2009, orphan designation (EU/3/09/653) was granted by the European Commission to Alexion Europe SAS, France, for eculizumab for the treatment of atypical haemolytic uraemic syndrome (aHUS).

Eculizumab in treatment of atypical haemolytic uraemic syndrome (aHUS) has been authorised in the EU as Soliris since 24 November 2011.

The sponsor’s address was updated in November 2019.

Key facts

Active substance
Eculizumab
Medicine name
Soliris
Intended use
Treatment of atypical haemolytic uraemic syndrome (aHUS)
Orphan designation status
Positive
EU designation number
EU/3/09/653
Date of designation
24/07/2009
Sponsor

Alexion Europe S.A.S.
103-105 Rue Anatole France
92300 Levallois Perret
France
E-mail: MedInfo@alexion.com

Review of designation

During its meeting of 5-7 October 2011, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/09/653 for Soliris (eculizumab) as an orphan medicinal product for the treatment of atypical haemolytic uraemic syndrome. The COMP assessed whether, at the time of addition of a new indication to the 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 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 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 addition of a new indication to the marketing authorisation: Soliris (eculizumab) for the treatment of atypical haemolytic uraemic syndrome (PDF/56.64 KB)

    Adopted

    First published: 21/12/2011
    Last updated: 21/12/2011
    EMA/COMP/792994/2011

  • 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:

    Related content

    How useful was this page?

    Add your rating
    Average
    1 rating
    1 rating