Vertex’s Trikafta Receives the US FDA’s Approval for the Treatment of Children with Cystic Fibrosis Aged 2 Through 5 Years
- The US FDA has approved the expanded use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in children aged 2-5yrs. with CF with 1 F508del mutation in the CFTR gene or a mutation in the CFTR gene responsive to Trikafta based on in vitro data
- The approval was based on the 24wk. two-part P-III trial evaluating Trikafta which showed that the therapy was well tolerated with a safety profile consistent with that observed in older age groups, improvement in sweat chloride concentration, a measure of CFTR function & lung function. The results were published in the AJRCCM
- The therapy was approved in the US, Canada, Switzerland, Australia, New Zealand & Israel, EU, the UK, Iceland, Liechtenstein & Norway as Kaftrio in combination with Kalydeco
Ref: Businesswire | Image: Vertex
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