On 29 May 2019, orphan designation (EU/3/19/2163) was granted by the European Commission to Voisin Consulting S.A.R.L., France, for N-(trans-3-(5-((R)-1-hydroxyethyl)-1,3,4-oxadiazol-2-yl)cyclobutyl)-3-phenylisoxazole-5-carboxamide (also known PTI-428) for the treatment of cystic fibrosis.
|Disease / condition||
Treatment of cystic fibrosis
|Date of first decision||
|EU designation number||
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.