Results from both laboratory testing and earlier human trials paved the way for an expanded indication of ivacaftor for treating cystic fibrosis.
Marketed as Kalydeco, ivacaftor already was approved to treat cystic fibrosis in patients aged 2 years or older with 1 of 10 specific mutations in the cystic fibrosis transmembrane conductance regulator gene. The new indication expands the number of treatable mutations to 33.
Voelker R. Expanded Cystic Fibrosis Therapy. JAMA. 2017;318(1):20. doi:10.1001/jama.2017.7643