New treatment targeting cause of CF approved

Ivacaftor (Kalydeco, Vertex) is indicated for people with CF, aged six and older, who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. 

In people with the G551D mutation, estimated to be around 250 in Australia (8% of CF patients), ivacaftor helps the defective CFTR protein function more normally.