The US Food and Drug Administration (FDA) has approved elexacaftor/tezacaftor/ivacaftor and ivacaftor (Trikafta®) for use in patients with cystic fibrosis (CF) aged 2 to 5 years who have at least 1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that might respond to elexacaftor/tezacaftor/ivacaftor and ivacaftor based on in vitro data, Vertex Pharmaceuticals announced.

“Early intervention with CFTR modulator therapies like Trikafta can offer the potential to improve the trajectory of CF lung disease,” said Jennifer Goralski, MD, assistant professor of medicine and pediatrics at University of North Carolina School of Medicine and a lead principal investigator in the Trikafta 2- to 5-year-old pivotal clinical trial. 

The researchers conducted a 24-week phase 3 open-label study on 75 children with CF aged 2 to 5 years to assess the safety, pharmacokinetics, and efficacy of the novel drug.

According to the results, the safety profile of elexacaftor/tezacaftor/ivacaftor and ivacaftor was similar to the one observed in older age groups, and all participants tolerated the medication well. Moreover, treatment with a combination of elexacaftor/tezacaftor/ivacaftor and ivacaftor resulted in improved sweat chloride concentration and lung function.

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The article detailing the study findings has recently been published in the American Journal of Respiratory and Critical Care Medicine.

Prior to the label expansion, the medication was approved by the FDA for use in individuals aged 6 years and older with CF caused by certain mutations, in the US, Canada, Switzerland, Australia, New Zealand, and Israel, as well as in the EU, the UK, Iceland, Liechtenstein, and Norway as ivacaftor/tezacaftor/elexacaftor (Kaftrio®) in a combination regimen with ivacaftor (Kalydeco®).

The global biotechnology company has also submitted applications for the use of the novel medication in children aged 2 to 5 to the European Medicines Agency and the Medicines and Healthcare Products Regulatory Agency.


Vertex announces US FDA Approval for Trikafta® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in children with cystic fibrosis ages 2 through 5 with certain mutations. News release. Vertex Pharmaceuticals Incorporated; April 26, 2023.