EU/3/19/2163

Table of contents

About

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.

Key facts

Active substance
N-(trans-3-(5-((R)-1-hydroxyethyl)-1,3,4-oxadiazol-2-yl)cyclobutyl)-3-phenylisoxazole-5-carboxamide
Disease / condition
Treatment of cystic fibrosis
Date of first decision
29/05/2019
Outcome
Positive
EU designation number
EU/3/19/2163

Sponsor's contact details

Voisin Consulting S.A.R.L.
64 Avenue Pierre Grenier
92100 Boulogne-Billancourt
France
Tel. +33 1413 16364
E-mail: orphan@voisinconsulting.com

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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