Elexacaftor/Ivacaftor/Tezacaftor: First Approval Sheridan M. Hoy 10.6084/m9.figshare.10317176.v1 https://adisjournals.figshare.com/articles/online_resource/Elexacaftor_Ivacaftor_Tezacaftor_First_Approval/10317176 <p>Compliance with Ethical Standards<b><br></b></p><p><i>Funding</i>: The preparation of this review was not supported by any external funding.<br></p> <p><i>Conflict of interest</i>: 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.<br></p><p><br></p><p>Additional information about this Adis Drug Review can be found <a href="http://www.springer.com/gp/adis/products-services/adis-journals-newsletters/adis-drug-reviews"><b>here</b></a>.<br></p><p><br></p><p>Abstract<br></p><p>A 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 (<i>F508del</i>). 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 <i>F508del </i>mutation in the <i>CFTR </i>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 <i>F508del </i>mutation in the <i>CFTR </i>gene.<br></p><p><br></p><p>© Springer Nature Switzerland AG 2019<br></p> 2019-12-01 18:00:47 Elexacaftor/Ivacaftor/Tezacaftor Trikafta™ Cystic Fibrosis Review AdisInsight Report