Vertex wins European approval for new cystic fibrosis combination

European regulators have approved Vertex’s new cystic fibrosis (CF) combination treatment Symkevi. The European Commission’s greenlight for Symkevi (tezacaftor/ivacaftor) will allow it to be used in combination with Kalydeco (ivacaftor) for patients aged 12-plus. In doing so it will offer a new treatment option for individuals with two copies of the F508del mutation, the most common mutation in cystic fibrosis. Symkevi has also been approved as a combination treatment for those who have one copy of the F508del mutation and a copy of one of 14 mutations in which the gene shows residual cystic fibrosis transmembrane conductance regulator (CFTR) activity. The EU marketing authorization was based on results from two phase 3 studies, which showed that treatment with tezacaftor/ivacaftor in combination with ivacaftor provides benefits across different CF populations, including statistically significant improvements in lung function.