On 30 May 2016, orphan designation (EU/3/16/1661) was granted by the European Commission to Alexion Europe SAS, France, for Fc- and CDR-modified humanised monoclonal antibody against C5 for the treatment of paroxysmal nocturnal haemoglobinuria.
Please note that this product (marketed as Ultomiris) was withdrawn from the Community Register of designated orphan medicinal products in June 2019 upon request of the marketing authorisation holder at the time of the granting of a marketing authorisation.
This medicine is now known as ravulizumab.
Fc- and CDR-modified humanised monoclonal antibody against C5 (ravulizumab)
|Disease / condition||
Treatment of paroxysmal nocturnal haemoglobinuria
|Date of first decision||
|EU designation number||
Review of designation
The sponsor formally requested the withdrawal of the orphan designation prior to the Committee’s final opinion. More information is available in the withdrawal assessment report – orphan maintenance .
Sponsor's contact details
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;