Vertex gains FDA approval for expanded use of CF drug Trikafta

27 Apr 2023
www.pharmaceutical-technology.com
Phase 3Drug ApprovalClinical Result
Vertex’s Trikafta is now approved to treat cystic fibrosis in children aged between two and five years with certain mutations. Credit: Jon Pauling from Pixabay.
Vertex PhTrikaftaicals has received approvacystic fibrosisood and Drug Administration (FDA) to expand the use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to treat cystic fibrosis (CF) in children aged between two and five years with certain mutations.
Vertex Pharmaceuticalsndicated for CF patients with a minFood and Drug Administration (FDA)e cystic fibrosis tranTrikaftae elexacaftor tezacaftor(ivacaftore, orivacaftorutation in cystic fibrosis (CF)esponds to Trikafta based on in vitro data.
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It was previously approved to treat CF in children aged between six and 11 years.
Trikafta is an oral medicineDocetaxelbeenSolid Tumoro boost the CFTR protein’s quantity and function at the cell surface.
Vertex Pharmaceuticals chief medical officer and global medicines development and medical affairs executive vice-president Carmen Bozic stated: “Since its initial approval, Trikafta has had a significant impact on the CF community, transforming the lives of thousands of people living with cystic fibrosis.
“We remain steadfast in our commitment to bringing highly effective CF treatments to people of all ages living with this disease.”
Trikaftalatory approval for the label expansion was supported byCFTR proteinpen-label Phase III trial conducted in 75 children with CF aged between two and five years.
Vertex Pharmaceuticalse tolerance of Trikafta and indicated that its safety profile was in line with that observed in older age groups.Trikaftacystic fibrosis
The regimen also showed improvements in sweat chloride concentration, which is a measure of CFTR function, and lung function.
Trikafta has already been approved in Switzerland, Canada, Australia, New Zealand, Israel and the US to treat CF in people aged six years and above with certain mutations.
It has also received approvals in theTrikaftan Union, Iceland, Liechtenstein, Norway and the UK as KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with KALYDECO (ivacaftor).
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