Current state of CFTR modulators for treatment of Cystic Fibrosis

Small molecular modulators of the cystic fibrosis transmembrane conductance regulator protein are transforming the care of people with cystic fibrosis. Highly effective modulators are now approved for nearly 90% of the adult CF population. They dramatically improve lung function, respiratory symptoms, and reduce pulmonary exacerbations. Recent efforts are expanding the availability of these therapies to a growing number of pediatric patients. The impact of modulators on extrapulmonary CF manifestations varies, although profound improvements in nutrition have been demonstrated. Observational studies and real-world research suggest that treatment benefits are sustained over time, and that maximal impact may be obtained with early use. The development of alternative approaches to restoring cystic fibrosis transmembrane conductance regulator (CFTR) function is needed for those with ineligible mutations.