EU/3/17/1828: Orphan designation for the treatment of cystic fibrosis

ivacaftor / tezacaftor


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
  • ivacaftor
  • tezacaftor
Intended use
Treatment of cystic fibrosis
Orphan designation status
EU designation number
Date of designation

Vertex Pharmaceuticals (Ireland) Limited
Unit 49
Northwood Court
Block F2 Santry
Dublin 9

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).

Documents related to this orphan designation evaluation

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:

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