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.

This medicine is now known as tezacaftor and ivacaftor.

Key facts

Active substance
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 (tezacaftor and ivacaftor)
Disease / condition
Treatment of cystic fibrosis
Date of decision
Orphan decision number

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.

Sponsor's contact details

Vertex Pharmaceuticals (Europe) Limited
2 Kingdom Street
London W2 6BD
United Kingdom
Telephone: +44 (0)20 3204 5235

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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