english.prescrire.org > Spotlight > Archives : 2023 > Ivacaftor + tezacaftor + elexacaftor (Kaftrio°) in cystic fibrosis with at least one delta F508 mutation from 6 years of age

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Ivacaftor + tezacaftor + elexacaftor (Kaftrio°) in cystic fibrosis with at least one delta F508 mutation from 6 years of age

 Marketing Authorisations  Triple therapy with the CFTR modulators ivacaftor + tezacaftor + elexacaftor was initially authorised in the European Union for adults and adolescents who have cystic fibrosis with a delta F508 mutation on at least one of the CFTR alleles. It has also been authorised for use in children in the same situation, aged 6 years and older.
Full article (2 pages) available for download by subscribers

In the European Union, triple therapy with the CFTR modulators ivacaftor + tezacaftor + elexacaftor was initially authorised for adults and adolescents aged 12 years and above, who have cystic fibrosis with a delta F508 mutation on at least one of the CFTR alleles, irrespective of the activity of the CFTR protein. In these patients, this combination is the first-choice drug based on its demonstrated symptomatic efficacy, albeit only in the short term, especially in alleviating respiratory symptoms (see  "Ivacaftor + tezacaftor + elexacaftor (Kaftrio°) in cystic fibrosis with at least one F508del mutation. Respiratory symptoms alleviated in the short term" Prescrire Int 235 >Pdf, subscribers only).

This treatment has also been authorised in the same situation for patients aged 6 years and older, and is available at half the dose strength.

©Prescrire 1 June 2023

Source: "Ivacaftor + tezacaftor + elexacaftor (Kaftrio°) in cystic fibrosis with at least one delta F508 mutation from 6 years of age" Prescrire International 2023; 32 (249): 151-152. Subscribers only.

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See also:

"Ivacaftor + tezacaftor + elexacaftor
(Kaftrio°) in cystic fibrosis
with at least one F508del mutation.
Respiratory symptoms alleviated
in the short term"
(March 2022)
Pdf, subscribers only


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