Cystic fibrosis drug approved for patients aged 6-11 years worked well in clinical practice.

Ivacaftor (IVA) and lumacaftor (LUM) modulate the cystic fibrosis (CF) transmembrane conductance regulator protein. IVA targets gating defects and LUM improves the conformational stability of the F508del mutation. This combined drug was approved for homozygous F508del CF patients aged 12 years or more in 2015 and was extended to children aged 6-11 in 2018.