EU/3/18/2117: Orphan designation for the treatment of cystic fibrosis
Ivacaftor, potassium(benzenesulfonyl)({[6-(3-{2-[1-(trifluoromethyl) cyclopropyl]ethoxy}-1H-pyrazol-1-yl)-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridin-3-yl]carbonyl})azanide, tezacaftor
Table of contents
Overview
On 14 December 2018, orphan designation (EU/3/18/2117) was granted by the European Commission to Vertex Pharmaceuticals (Europe) Limited, United Kingdom, for ivacaftor, potassium(benzenesulfonyl)({[6-(3-{2-[1-(trifluoromethyl) cyclopropyl]ethoxy}-1H-pyrazol-1-yl)-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridin-3-yl]carbonyl})azanide, tezacaftor for the treatment of cystic fibrosis.
The sponsorship was transferred to Vertex Pharmaceuticals (Ireland) Limited, Ireland, in February 2019.
The sponsor’s address was updated in January 2022.
Key facts
Active substance |
Ivacaftor, potassium(benzenesulfonyl)({[6-(3-{2-[1-(trifluoromethyl) cyclopropyl]ethoxy}-1H-pyrazol-1-yl)-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridin-3-yl]carbonyl})azanide, tezacaftor
|
Intended use |
Treatment of cystic fibrosis
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/18/2117
|
Date of designation |
14/12/2018
|
Sponsor |
Vertex Pharmaceuticals (Ireland) Limited |
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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: