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Please note that this product (marketed as Ultomiris) was withdrawn from the Community Register of designated orphan medicinal products in June 2019 upon request of the marketing authorisation holder at the time of the granting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.

Key facts

Active substance
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 PDF iconwithdrawal assessment report – orphan maintenance .

The Committee for Orphan Medicinal Products reviewed the orphan designation of Ultomiris at the time of marketing authorisation

Sponsor's contact details

Alexion Europe SAS
1-15, avenue Edouard Belin
92500 Rueil-Malmaison
Tel. +33 1 47 32 36 03
Fax +33 1 47 10 06 11

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;

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