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.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in July 2021 on request of the Sponsor.
Treatment of cystic fibrosis
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Documents related to this orphan designation evaluation
EU/3/19/2163: Public summary of opinion on orphan designation: N-(trans-3-(5-((R)-1-hydroxyethyl)-1,3,4-oxadiazol-2-yl)cyclobutyl)-3-phenylisoxazole-5-carboxamide for the treatment of cystic fibrosis (PDF/226.79 KB)
First published: 09/08/2019
Last updated: 30/07/2021
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.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: