On 17 October 2003, orphan designation (EU/3/03/166) was granted by the European Commission to QuadraMed, United Kingdom, for eculizumab for the treatment of paroxysmal nocturnal haemoglobinuria.
The sponsorship was transferred to Alexion Europe SAS, France, in March 2006.
Eculizumab in treatment of paroxysmal nocturnal haemoglobinuria has been authorised in the EU as Soliris since 20 June 2007.
The sponsor’s address was updated in November 2019.
Treatment of paroxysmal nocturnal haemoglobinuria
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Documents related to this orphan designation evaluation
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: