Long-term safety and efficacy of lumacaftor–ivacaftor therapy in children aged 6–11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study

Long-term data from two Phase III studies (n=239; 90% completed 96 weeks) show lumacaftor–ivacaftor treatment was generally well-tolerated; 99% had mild or moderate adverse effects, consistent with its known safety profile. Efficacy was generally maintained.

Source:

The Lancet Respiratory Medicine