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FDA Expands Indication for Vertex Pharmaceuticals Cystic Fibrosis Drug

NEW YORK – The US Food and Drug Administration has approved Vertex Pharmaceuticals' cystic fibrosis drug Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for younger patients with certain mutations, the biotechnology company said Wednesday. 

In 2021, Boston-based Vertex's Trikafta was approved for cystic fibrosis patients ages 6 to 11 years with certain CFTR gene variants, including F508del mutations. The FDA's latest approval expands the drug's use to children with cystic fibrosis aged 2 to 5 years with those same genetic mutations.

Trikafta is an oral medication designed to increase the quantity and function of the CFTR protein at the cell surface in patients with cystic fibrosis, a rare disease caused by various mutations in the CFTR gene.

The label expansion was based on data from a Phase III open-label study that enrolled 75 cystic fibrosis patients 2 to 5 years of age. The drug improved sweat chloride concentration and lung function in patients, according to data published in the American Journal of Respiratory and Critical Care Medicine, and Trikafta's safety profile was consistent with what has been observed in older age groups.

Vertex has submitted applications with regulators in other countries seeking approval of the drug in younger cystic fibrosis patients, including in the European Union and the UK.