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 Pharmaceuticals has received approval from the US Food 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.

The treatment is now indicated for CF patients with a minimum of one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or with a mutation in the CFTR gene that responds 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 medicine and has been designed to 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.”

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The regulatory approval for the label expansion was supported by a 24-week open-label Phase III trial conducted in 75 children with CF aged between two and five years.

The trial showcased the tolerance of Trikafta and indicated that its safety profile was in line with that observed in older age groups.

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 the European Union, Iceland, Liechtenstein, Norway and the UK as KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with KALYDECO (ivacaftor).