Cystic fibrosis: triple drug regimens that target defective ion channel improve lung function, studies show

Triple drug combinations that improve the activity of the cystic fibrosis transmembrane conductance regulator (CFTR), the anion channel that is defective or deficient in the condition, significantly improve lung function in patients with cystic fibrosis who have the most common CFTR gene mutations, two early stage randomised trials have reported.12

“These reports represent a major breakthrough in cystic fibrosis therapeutics, with the potential for improving health and possibly survival in all patients who carry the most common CFTR mutation,” said Fernando Holguin of the University of Colorado in Aurora, USA, in an accompanying editorial.3

Both trials evaluated the efficacy and safety of one of two new generation, small molecule CFTR correctors, VX-659 and VX-445. These compounds …