Cystic Fibrosis Ireland is pleased to welcome the European Commission decision to approve the label extension of Kaftrio®(ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in patients ages 6 through 11 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. There remains a few more procedural steps to be undertaken in Ireland before it becomes available to young people with CF in the 6-11 age cohort, but this announcement marks a further welcome extension of Kaftrio and is consistent with the Portfolio/‘Orkambi' deal of 2017 that was supported by Cystic Fibrosis Ireland. This decision follows on from the positive recommendation of the CHMP Committee for the European Medicines Agency (EMA) previously reported by CFI on 17th November 2021. CFI will keep you updated on when this important drug therapy becomes available in Ireland - hopefully before the summer of 2022. As with all therapies it is important that those families who have the potential to benefit from this drug therapy should discuss their suitability with their clinician, including benefits and possible side effects.   

Please find press release from Vertex  Pharmaceuticals (Europe) here