EU/3/09/653: Orphan designation for the treatment of atypical haemolytic uraemic syndrome (aHUS)
Table of contents
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.
Treatment of atypical haemolytic uraemic syndrome (aHUS)
|Orphan designation status||
|EU designation number||
|Date of designation||
Alexion Europe S.A.S.
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
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)
First published: 21/12/2011
Last updated: 21/12/2011
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: