On 12 December 2017, orphan designation (EU/3/17/1946) was granted by the European Commission to FGK Representative Service GmbH, Germany, for (2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidine-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridine-2-yl)-4-fluoropyrrolidine-2-carboxamide (also known as ACH-0144471) for the treatment of paroxysmal nocturnal haemoglobinuria.

The sponsorship was transferred to  Alexion Europe S.A.S. France, in June 2020.

Key facts

Active substance
Disease / condition
Treatment of paroxysmal nocturnal haemoglobinuria
Date of first decision
EU designation number

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

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

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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