EU/3/17/1828
Table of contents
About
On 27 February 2017, orphan designation (EU/3/17/1828) was granted by the European Commission to Vertex Pharmaceuticals (Europe) Limited, United Kingdom, for 1-(2,2-difluoro-2H-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropane-1-carboxamide (also known as tezacaftor) and ivacaftor for the treatment of cystic fibrosis.
1-(2,2-difluoro-2H-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropane-1-carboxamide and ivacaftor has been authorised in the EU as Symkevi since 31 October 2018.
The sponsorship was transferred to Vertex Pharmaceuticals (Ireland) Limited, Ireland, in December 2018.
This medicine is now known as tezacaftor and ivacaftor.
Key facts
Disease / condition |
Treatment of cystic fibrosis
|
Date of first decision |
27/02/2017
|
Outcome |
Positive
|
EU designation number |
EU/3/17/1828
|
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
The Committee for Orphan Medicinal Products reviewed the orphan designation of Symkevi at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.
More information is available in the Symkevi : Orphan maintenance assessment report (initial 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.