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.
EU/3/17/1946: Public summary of opinion on orphan designation: (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 for the treatment of p... (PDF/130.78 KB)
First published: 24/01/2018
Last updated: 24/01/2018
|Disease / condition||
Treatment of paroxysmal nocturnal haemoglobinuria
|Date of first decision||
|EU designation number||
Review of designation
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.