Please note that this product (marketed as Orkambi) was withdrawn from the Community Register of designated orphan medicinal products in October 2015 upon request of the marketing authorisation holder at the time of the granting of a marketing authorisation.
On 22 August 2014, orphan designation (EU/3/14/1333) was granted by the European Commission to Vertex Pharmaceuticals (U.K.) Limited, United Kingdom, for lumacaftor/ivacaftor for the treatment of cystic fibrosis.
|Disease / condition||
Treatment of cystic fibrosis
|Date of first decision||
|EU designation number||
Review of designation
Sponsor's contact details
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.