CFI is pleased to report that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the label extension for Kaftrio, for the treatment of children with cystic fibrosis (CF) ages two through five years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Please note that a CHMP positive opinion is not a Marketing Authorisation decision, but its is an important step towards such authorisation. This means that the label extension is not yet licensed in the European Union, but is likely to be in the next few weeks. The standard process according to the EU legislation is that Marketing Authorisation decisions are taken by the European Commission.

Kaftrio for two to five year olds (who are eligible) will be available in Ireland once approved by the European Commission and then the HSE. CFI does not anticipate any barriers or undue delays to availability to eligible children in Ireland at this stage. Our rough estimate is availability by January 2024, but we will update you as soon as we know more.