EU/3/19/2163: Orphan designation for the treatment of cystic fibrosis


Table of contents


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Please note that this product was withdrawn from the Union Register of orphan medicinal products in July 2021 on request of the Sponsor.

Key facts

Active substance
Intended use
Treatment of cystic fibrosis
Orphan designation status
EU designation number
Date of designation

Voisin Consulting S.A.R.L.
64 Avenue Pierre Grenier
92100 Boulogne-Billancourt
Tel. +33 1413 16364

Documents related to this orphan designation evaluation

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.

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:

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