The French compassionate programme of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with advanced lung disease and no F508del CFTR variant

The European Medicines Agency has approved the cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination elexacaftor/tezacaftor/ivacaftor (ETI) for people with cystic fibrosis (CF) carrying at least one F508del variant. The United States Food and Drug Administration (FDA) also...

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Published in:The European respiratory journal Vol. 61; no. 5
Main Authors: Burgel, Pierre-Régis, Sermet-Gaudelus, Isabelle, Durieu, Isabelle, Kanaan, Reem, Macey, Julie, Grenet, Dominique, Porzio, Michele, Coolen-Allou, Nathalie, Chiron, Raphael, Marguet, Christophe, Douvry, Benoit, Dufeu, Nadine, Danner-Boucher, Isabelle, Foucaud, Pierre, Lemonnier, Lydie, Girodon, Emmanuelle, Da Silva, Jennifer, Martin, Clémence, Andrejak, Claire, Priou, Pascaline, Richaud-Thiriez, Bénédicte, Montcouquiol, Sylvie, Remus, Natacha, Fanton, Annlyse, Gachelin, Elsa, Vuillard, Constance, Audousset, Camille, Duthoit, Louise, Bellet-Fraysse, Elisabeth, Languepin, Jeanne, Nove-Josserand, Raphaele, Ohlmann, Camille, Raynaud, Quitterie, Billon, Yves, Tissot, Adrien, Leroy, Sylvie, Aubourg, Frédérique, Burnet, Espérie, Carlier, Nicolas, Fajac, Isabelle, Honoré, Isabelle, Le Bourgeois, Muriel, Le Clainche-Viala, Laurence, Corvol, Harriet, Dehillotte, Clémence, Brinchault, Graziella, Ravoninjatovo, Bruno, Le Bihan, Jean, Ramel, Sophie, Weiss, Laurence, de Miranda, Sandra, Cosson, Laure, Mankikian, Julie, Pouradier, Delphine
Format: Journal Article
Language:English
Published: England 01.05.2023
ISSN:1399-3003, 1399-3003
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Abstract The European Medicines Agency has approved the cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination elexacaftor/tezacaftor/ivacaftor (ETI) for people with cystic fibrosis (CF) carrying at least one F508del variant. The United States Food and Drug Administration (FDA) also approved ETI for people with CF carrying one of 177 rare variants. An observational study was conducted to evaluate the effectiveness of ETI in people with CF with advanced lung disease who were not eligible for ETI in Europe. All patients with no F508del variant and advanced lung disease (defined as having a percent predicted forced expiratory volume (ppFEV ) <40% and/or being under evaluation for lung transplantation) and enrolled in the French compassionate programme initiated ETI at recommended doses. Effectiveness was evaluated by a centralised adjudication committee at 4-6 weeks in terms of clinical manifestations, sweat chloride concentration and ppFEV . Among the first 84 people with CF included in the programme, ETI was effective in 45 (54%), and 39 (46%) were considered to be nonresponders. Among the responders, 22 (49%) out of 45 carried a variant that is not currently approved by the FDA for ETI eligibility. Important clinical benefits, including suspending the indication for lung transplantation, a significant decrease in sweat chloride concentration by a median (interquartile range (IQR)) -30 (-14--43) mmol·L (n=42; p<0.0001) and an improvement in ppFEV by +10.0 (6.0-20.5) percentage points (n=44; p<0.0001), were observed in those for whom treatment was effective. Clinical benefits were observed in a large subset of people with CF with advanced lung disease and variants not currently approved for ETI.
AbstractList 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.BACKGROUNDThe 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.An observational study was conducted to evaluate the effectiveness of ETI in pwCF with advanced lung disease that were not eligible to ETI in Europe. All patients with no F508del variant and advanced lung disease (defined as having a percent predicted forced expiratory volume (ppFEV1)<40 and/or being under evaluation for lung transplantation) and enrolled in the French Compassionate Program initiated ETI at recommended doses. Effectiveness was evaluated by a centralized adjudication committee at 4-6 weeks in terms of clinical manifestations, sweat chloride concentration and ppFEV1.METHODSAn observational study was conducted to evaluate the effectiveness of ETI in pwCF with advanced lung disease that were not eligible to ETI in Europe. All patients with no F508del variant and advanced lung disease (defined as having a percent predicted forced expiratory volume (ppFEV1)<40 and/or being under evaluation for lung transplantation) and enrolled in the French Compassionate Program initiated ETI at recommended doses. Effectiveness was evaluated by a centralized adjudication committee at 4-6 weeks in terms of clinical manifestations, sweat chloride concentration and ppFEV1.Among the first 84 pwCF included in the program, ETI was effective in 45 (54%) and 39 (46%) were considered to be non-responders. Among the responders 22/45 (49%) carried a CFTR variant that is not currently approved by FDA for ETI eligibility. Important clinical benefits, including suspending the indication for lung transplantation, a significant decrease in sweat chloride concentration by a median [IQR] -30 [-14;-43]mmol·l-1 (n=42; p<0.0001) and an improvement in ppFEV1 by+10.0 [6.0; 20.5] (n=44, p<0.0001), were observed in those for whom treatment was effective.RESULTSAmong the first 84 pwCF included in the program, ETI was effective in 45 (54%) and 39 (46%) were considered to be non-responders. Among the responders 22/45 (49%) carried a CFTR variant that is not currently approved by FDA for ETI eligibility. Important clinical benefits, including suspending the indication for lung transplantation, a significant decrease in sweat chloride concentration by a median [IQR] -30 [-14;-43]mmol·l-1 (n=42; p<0.0001) and an improvement in ppFEV1 by+10.0 [6.0; 20.5] (n=44, p<0.0001), were observed in those for whom treatment was effective.Clinical benefits were observed in a large subset of pwCF with advanced lung disease and CFTR variants not currently approved for ETI.CONCLUSIONClinical benefits were observed in a large subset of pwCF with advanced lung disease and CFTR variants not currently approved for ETI.
The European Medicines Agency has approved the cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination elexacaftor/tezacaftor/ivacaftor (ETI) for people with cystic fibrosis (CF) carrying at least one F508del variant. The United States Food and Drug Administration (FDA) also approved ETI for people with CF carrying one of 177 rare variants. An observational study was conducted to evaluate the effectiveness of ETI in people with CF with advanced lung disease who were not eligible for ETI in Europe. All patients with no F508del variant and advanced lung disease (defined as having a percent predicted forced expiratory volume (ppFEV ) <40% and/or being under evaluation for lung transplantation) and enrolled in the French compassionate programme initiated ETI at recommended doses. Effectiveness was evaluated by a centralised adjudication committee at 4-6 weeks in terms of clinical manifestations, sweat chloride concentration and ppFEV . Among the first 84 people with CF included in the programme, ETI was effective in 45 (54%), and 39 (46%) were considered to be nonresponders. Among the responders, 22 (49%) out of 45 carried a variant that is not currently approved by the FDA for ETI eligibility. Important clinical benefits, including suspending the indication for lung transplantation, a significant decrease in sweat chloride concentration by a median (interquartile range (IQR)) -30 (-14--43) mmol·L (n=42; p<0.0001) and an improvement in ppFEV by +10.0 (6.0-20.5) percentage points (n=44; p<0.0001), were observed in those for whom treatment was effective. Clinical benefits were observed in a large subset of people with CF with advanced lung disease and variants not currently approved for ETI.
Author Richaud-Thiriez, Bénédicte
Montcouquiol, Sylvie
Audousset, Camille
Mankikian, Julie
Fanton, Annlyse
Ohlmann, Camille
Cosson, Laure
Remus, Natacha
Durieu, Isabelle
Sermet-Gaudelus, Isabelle
Macey, Julie
Fajac, Isabelle
Da Silva, Jennifer
Porzio, Michele
Gachelin, Elsa
Kanaan, Reem
Duthoit, Louise
Honoré, Isabelle
Dehillotte, Clémence
Le Clainche-Viala, Laurence
de Miranda, Sandra
Tissot, Adrien
Le Bourgeois, Muriel
Girodon, Emmanuelle
Corvol, Harriet
Burnet, Espérie
Leroy, Sylvie
Ramel, Sophie
Priou, Pascaline
Martin, Clémence
Raynaud, Quitterie
Aubourg, Frédérique
Bellet-Fraysse, Elisabeth
Burgel, Pierre-Régis
Lemonnier, Lydie
Grenet, Dominique
Le Bihan, Jean
Danner-Boucher, Isabelle
Chiron, Raphael
Billon, Yves
Ravoninjatovo, Bruno
Marguet, Christophe
Coolen-Allou, Nathalie
Douvry, Benoit
Languepin, Jeanne
Pouradier, Delphine
Carlier, Nicolas
Foucaud, Pierre
Vuillard, Constance
Dufeu, Nadine
Andrejak, Claire
Weiss, Laurence
Nove-Josserand, Raphaele
Brinchault, Graziella
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BackLink https://www.ncbi.nlm.nih.gov/pubmed/36796836$$D View this record in MEDLINE/PubMed
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ContentType Journal Article
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Copyright_xml – notice: Copyright ©The authors 2023. For reproduction rights and permissions contact permissions@ersnet.org.
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References 38423591 - Eur Respir J. 2024 Feb 29;63(2):2400233. doi: 10.1183/13993003.00233-2024
38423592 - Eur Respir J. 2024 Feb 29;63(2):2301392. doi: 10.1183/13993003.01392-2023
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