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.

“The approval of ivacaftor in Australia is an important milestone for the cystic fibrosis community,” said Simon Bedson, Senior Vice President of International Commercial Operations for Vertex.

In two global phase 3 studies of people with CF who have at least one copy of the G551D mutation those treated with ivacaftor experienced significant and sustained improvements in lung function and weight gain compared to placebo.

In one study, adults and adolescents who took ivacaftor were also found to be significantly less likely to experience pulmonary exacerbations,