European regulators have approved Vertex’s new cystic fibrosis (CF) combination treatment tezacaftor/ivacaftor (marketed as Symkevi). Tezacaftor/ivacaftor in combination with ivacaftor was approved by the US FDA in February 2018 and is marketed as Symdeko.
The European Commission’s greenlight for Symkevi 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.