Basic Information
Kaftrio
Regulatory Information
EMEA/H/C/005269
August 21, 2020
June 25, 2020
27
April 10, 2025
Company Information
Ireland
Unit 49, Block 5 Northwood Court Northwood Crescent Dublin 9 D09 T665
VERTEX PHARMACEUTICALS (IRELAND) LIMITED
Drug Classification
Active Substances Detail
EMA Resources
Detailed Information
Therapeutic Indication
### Therapeutic indication Kaftrio is indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Kaftrio granules are indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in paediatric patients aged 2 to less than 6 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Overview Summary
Kaftrio is a medicine used to treat patients aged 2 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 two 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 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 can be found here: [https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182117](https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182117) Kaftrio contains the active substances ivacaftor, tezacaftor and elexacaftor.