EU/3/18/2117

About

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.

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
Disease / condition
Treatment of cystic fibrosis
Date of first decision
14/12/2018
Outcome
Positive
EU designation number
EU/3/18/2117

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Vertex Pharmaceuticals (Ireland) Limited
28-32 Pembroke Street Upper
Dublin 2
D02 EK84
Ireland
Tel. +353 1761 7296
E-mail: vertexmedicalinfo@vrtx.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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