ivacaftor / tezacaftor / elexacaftor

This medicine is authorised for use in the European Union.


Kaftrio is a medicine used to treat patients aged 12 years and above who have cystic fibrosis, an inherited disease that has severe effects on the lungs, the digestive system and other organs.

Cystic fibrosis can be caused by various mutations (changes) in the gene for a protein called ‘cystic fibrosis transmembrane conductance regulator’ (CFTR). People have 2 copies of this gene, one inherited from each parent and the disease only occurs when there is a mutation in both copies.

Kaftrio is used in combination with ivacaftor (150 mg) in patients whose cystic fibrosis is due to at least one F508del mutation in the CFTR gene.

Cystic fibrosis is rare, and Kaftrio was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 14 December 2018. Further information on the orphan designation is available.

Kaftrio contains the active substances ivacaftor, tezacaftor and elexacaftor.

This EPAR was last updated on 25/05/2021

Authorisation details

Product details
Agency product number
Active substance
  • ivacaftor
  • tezacaftor
  • elexacaftor
International non-proprietary name (INN) or common name
  • ivacaftor
  • tezacaftor
  • elexacaftor
Therapeutic area (MeSH)
Cystic Fibrosis
Anatomical therapeutic chemical (ATC) code
Additional monitoringAdditional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.


This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Publication details
Marketing-authorisation holder
Vertex Pharmaceuticals (Ireland) Limited
Date of issue of marketing authorisation valid throughout the European Union
Contact address

28-32 Pembroke Street Upper
Dublin 2
D02 EK84

Product information

26/04/2021 Kaftrio - EMEA/H/C/005269 - II/0001


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Pharmacotherapeutic group

Other respiratory system products

Therapeutic indication

Kaftrio is indicated in a combination regimen with ivacaftor 150 mg tablets for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Assessment history

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