Phase III results from two studies of Trikafta for cystic fibrosis published in The New England Journal of Medicine and The Lancet.- Vertex Pharma
Vertex Pharmaceuticals Incorporated announced the concurrent publication in The New England Journal of Medicine (NEJM) and The Lancet of results from two Phase III studies of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF) in people ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation.
Data highlighting primary and secondary endpoints from a 24-week Phase III study in 403 people with one F508del mutation and one minimal function mutation (F/MF) in the CFTR gene were published in NEJM. In addition, The Lancet published data highlighting primary and secondary endpoints from a 4-week Phase III study in 107 people with two F508del mutations (F/F). Both studies met primary and all key secondary endpoints, demonstrating statistically significant and clinically meaningful improvements in lung function and other measures of disease.
In these studies, Trikafta was generally well tolerated. Detailed outcomes from these studies were previously communicated in May 2019. Data published in both NEJM and The Lancet contain additional analyses for these studies, including subgroup analyses, distribution of responses and safety.
See- "Elexacaftor�Tezacaftor�Ivacaftor for Cystic Fibrosis with a Single Phe508del Allel"- Peter G. Middleton, M.D., Marcus A. Mall, M.D., Pavel D?ev�nek, M.D., et al., for the VX17-445-102 Study Group-October 31, 2019 DOI: 10.1056/NEJMoa1908639.
See- "Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial."- Prof Harry G M Heijerman, MD Edward F McKone, MD , Damian G Downey, MD,et al. Published:October 31, 2019DOI:https://doi.org/10.1016/S0140-6736(19)32597-8.
Comment: this triplet drug combination can treat an estimated 90% of people with the genetic disease.
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