FDA approves expanded use of Trikafta in children with cystic fibrosis
Vertex Pharmaceuticals announced the FDA approved the expanded use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 2 through 5 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive to Trikafta based on in vitro data
Trikafta was previously approved by the FDA for use in people with CF 6 years and older with at least one F508del mutation or a mutation in the CFTR gene that is responsive to Trikafta based on in vitro data.
This label expansion was supported by a 24-week Phase III open-label study which enrolled 75 children ages 2 through 5 years old with CF to evaluate the safety, pharmacokinetics and efficacy of Trikafta. The regimen was generally well tolerated, with a safety profile consistent with that observed in older age groups, and led to improvements in sweat chloride concentration, a measure of CFTR function, and lung function. The data from this study were recently published in the American Journal of Respiratory and Critical Care Medicine.
See; "Phase III Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2 Through 5 Years with Cystic Fibrosis and at Least One F508del Allele"-Jennifer L. Goralski , Jordana E Hoppe , Marcus A Mall ,et al, https://doi.org/10.1164/rccm.202301-0084OC .PubMed: 36921081.
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