WS10.01 The French Compassionate Programme of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with advanced lung disease and no F508del CFTR variant

P.-R Burgel,I Sermet-Gaudelus,I Durieu,R Kanaan,J Macey,D Grenet,M Porzio,N Coolen-Allou,R Chiron,C Marguet,B Douvry,N Dufeu,I Danner-Boucher,P Foucaud,L Lemonnier,E Girodon,J Da Silva,C Martin

doi:10.1016/s1569-1993(23)00241-2

Abstract

Objective: The European Medicines Agency has approved the cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination elexacaftor-tezacaftor-ivacaftor (ETI) for people with cystic fibrosis (pwCF) carrying at least one F508del variant. The United States Food and Drug Administration (FDA) also approved ETI for pwCF carrying one of 177 rare variants. Our objective was to evaluate ETI in pwCF with advance lung disease and no F508del variant.

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