EU/3/17/1828: Orphan designation for the treatment of cystic fibrosis
ivacaftor / tezacaftor
Table of contents
Overview
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.
The sponsor’s address was updated in January 2022.
Key facts
Active substance |
|
Intended use |
Treatment of cystic fibrosis
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/17/1828
|
Date of designation |
27/02/2017
|
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.
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).
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: