Vertex Pharmaceuticals applies at EMA for expanded indication of Kaftrio to treat cystic fibrosis in patients ages 12 years and older. .

Vertex Pharmaceuticals Incorporated announced the European Medicines Agency (EMA) has validated a Type II Variation Marketing Authorization Application (MAA) for the expanded indication of Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor to treat CF in patients ages 12 years and older with at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. If approved, eligible patients who have one copy of the F508del mutation and another CFTR mutation, such as a gating (F/G) or residual function (F/RF) mutation, will also be eligible for treatment. The MAA is supported by positive results from the global Phase III study (445-104) with Kaftrio announced in July 2020. The application will now be reviewed by the Committee for Medicinal Products for Human Use (CHMP), which will issue an opinion to the European Commission regarding the potential approval for these patients.