Vertex Announces Publication in The New England Journal of Medicine of Phase 3 Results for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in People With Cystic Fibrosis

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[{"type":"text","content":" BOSTON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced publication in The New England Journal of Medicine (NEJM) of results from a Phase 3 study of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in people with cystic fibrosis (CF) ages 12 years and older who have one copy of the F508del mutation and one gating (F/G) or residual function (F/RF) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The manuscript includes data on primary and key secondary endpoints, which were previously reported and showed statistically significant and clinically meaningful improvements in lung function and sweat chloride, when compared to active control (either ivacaftor or tezacaftor/ivacaftor), as well as more detailed efficacy and safety data, including subgroup efficacy analyses.\n\n“This study is the third of three Phase 3 clinical trials in the TRIKAFTA program in the 12 years and older age group. Consistent with the prior outcomes, these results show clinically meaningful improvements in pulmonary function, sweat chloride and Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain scores,\" said Carmen Bozic, M.D., Executive Vice President and Chief Medical Officer, Vertex. “These results are especially notable given that all patients were treated with a CFTR modulator prior to initiating TRIKAFTA.”\n\n“The outcomes within this study, in particular those from the subgroup efficacy analysis by F/G and F/RF, are remarkable because they demonstrate additional benefit on top of standard of care and build further confidence for clinicians to treat people with CF who may have these mutations,” said Steven Rowe, M.D., Director, Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham.\n\nStudy 445-104 \n\nThe data published today are from a global Phase 3, randomized, double-blind, parallel-group study. All patients had a 4-week run-in period of either ivacaftor or tezacaftor/ivacaftor. Following the run-in, 258 patients were randomized to receive TRIKAFTA® or to remain on their prior regimen of ivacaftor or tezacaftor/ivacaftor for 8 weeks. Baseline was measured at the end of the run-in period, prior to the start of the 8-week treatment period. TRIKAFTA® improved the percent predicted forced expiratory volume in 1 second (pp...

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