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 patients whose cystic fibrosis is due to the F508del mutation inherited from one or both parents. If they have inherited this mutation from only one parent, they should also have another mutation called a ‘minimal function mutation’ from the other parent.

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.

The public assessment report and the orphan maintenance assessment report are in preparation and will be published under ‘Assessment history’ in the coming weeks.

This EPAR was last updated on 21/08/2020

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

Kaftrio - EMEA/H/C/005269 -


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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 are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or heterozygous for F508del in the CFTR gene with a minimal function (MF) mutation.

Assessment history

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