EU/3/03/166: Orphan designation for the treatment of paroxysmal nocturnal haemoglobinuria


Table of contents


Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in June 2019 at the end of the 12-year period of market exclusivity.

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.

Key facts

Active substance
Medicine name
Intended use
Treatment of paroxysmal nocturnal haemoglobinuria
Orphan designation status
EU designation number
Date of designation

Alexion Europe S.A.S.
103-105 Rue Anatole France
92300 Levallois Perret

Documents related to this orphan designation evaluation

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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