Elexacaftor/Ivacaftor/Tezacaftor: First Approval
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Funding: The preparation of this review was not supported by any external funding.
Conflict of interest: Sheridan Hoy is a salaried employee of Adis International Ltd/Springer
Nature, is responsible for the article
content and declares no relevant conflicts of interest.
information about this Adis Drug Review can be found here.
fixed-dose combination tablet of the cystic
fibrosis transmembrane conductance regulator (CFTR) corrector tezacaftor and the CFTR potentiator
ivacaftor with the next-generation CFTR corrector elexacaftor (hereafter
referred to as elexacaftor/ivacaftor/tezacaftor) [Trikafta™] has been developed
by Vertex Pharmaceuticals Inc. to treat patients with the most common cystic
fibrosis mutation (F508del). Its use has been associated with statistically significant and/or clinically meaningful improvements in lung
function and respiratory-related quality of life compared with comparator
regimens (placebo or ivacaftor/tezacaftor) in
multinational phase II and III studies, and in October 2019 elexacaftor/ivacaftor/tezacaftor was approved by
the US FDA for the treatment of cystic fibrosis in patients aged ≥ 12 years who
have ≥ 1 F508del mutation in the CFTR gene. A regulatory assessment for elexacaftor/ivacaftor/
tezacaftor as a treatment for cystic fibrosis is underway in the EU. This
article summarizes the milestones in the development of elexacaftor/ivacaftor/tezacaftor
leading to first approval for the treatment of cystic fibrosis in
patients aged ≥ 12 years who have ≥ 1 F508del mutation in the CFTR gene.
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