Gathering real-world compassionate data to expand eligibility for elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with N1303K or other rare<i>CFTR</i>variants: a viewpoint
Authors: Pierre-Régis Burgel, Isabelle Sermet-Gaudelus, Emmanuelle Girodon, Reem Kanaan, Jean Le Bihan, Natascha Remus, Bruno Ravoninjatovo, Dominique Grenet, Michele Porzio, Véronique Houdouin, Laurence Le Clainche-Viala, Isabelle Durieu, Raphaele Nove-Josserand, Jeanne Languepin, Bérangère Coltey, Anne Guillaumot, Camille Audousset, Raphaël Chiron, Laurence Weiss, Isabelle Fajac, Jennifer Da Silva, Clémence Martin
Journal: European Respiratory Journal (2024)

Elexacaftor-tezacaftor-ivacaftor (ETI) was approved in 2019 by the United States Food and Drug Administration (FDA) and in 2020 by the European Medicines Agency (EMA) for people with cystic fibrosis (pwCF). It is a combination of small molecules that bind to the defective cystic fibrosis transmembrane conductance regulator (CFTR) protein, thus allowing the rescue of CFTR structure and function [1]. Footnotes This manuscript has recently been accepted for publication in the European Respiratory Journal . It is published here in its accepted form prior to copyediting and typesetting by our production team. After these production processes are complete and the authors have approved the resulting proofs, the article will move to the latest issue of the ERJ online. Please open or download the PDF to view this article. Conflict of interest: Pierre-Régis Burgel reports support for the present manuscript from Association Vaincre la Mucoviscidose, Société Française de la Mucoviscidose, Filière Maladie Rare Muco CFTR. In addition, Pierre-Régis Burgel reports grants from Vertex Pharmaceuticals, GSK; consulting fees from Astra Zeneca, Chiesi, GSK, Insmed, Vertex, Viatris, Zambon; travel support Astra Zeneca, Chiesi; outside the submitted work. Conflict of interest: Isabelle Sermet-Gaudelus reports support for the present manuscript from Vertex Therapeutics, Tavanta. In addition, Isabelle Sermet-Gaudelus reports grants from Vertex Therapeutics, Tavanta; outside the submitted work. Conflict of interest: Isabelle Durieu and Jeanne Languepin report travel support from Mylan, outside the submitted work. Conflict of interest: Anne Guillaumot reports travel support from Asten, Boehringer Ingelheim, GSK, LFB, CSL Behring, Roche, Menarini; outside the submitted work. Conflict of interest: Camille Audousset reports travel support from Zambon, Viatris; advisory board participation from Vertex Pharmaceuticals; outside the submitted work. Conflict of interest: Raphaël Chiron reports travel support from ECFC; advisory board participation with Zambon, Vertex; outside the submitted work. Conflict of interest: Laurence Weiss reports travel support from Viatris; advisory board participation from Vertex; outside the submitted work. Conflict of interest: Isabelle Fajac reports grants from AbbVie, Bayer, Boehringer Ingelheim, Insmed, GSK, Vertex Pharmaceuticals, Zambon; consulting fees from AbbVie, Boehringer Ingelheim, Kither Biotech, Vertex Pharmaceuticals; leadership role with European Cystic Fibrosis Society; outside the submitted work. Conflict of interest: Clémence Martin reports lecture honoraria from Chiesi, AstraZeneca, Boehringer, GSL; travel support from Chiesi, Boehringer; outside the submitted work. Conflict of interest: All other authors have nothing to disclose.