ABSTRACT
CFTR pharmacotherapy has certainly been the success story of recent years. Leading the eld in terms of development phase is ivacaor, a CFTR potentiator, initially developed for and studied in patients carrying at least one copy of the class III mutation, G551D. Results from clinical trials have been impressive: treatment not only improved measures of CFTR function such as nasal potential dierence (nPD)3 and sweat chloride but, much more importantly, had signicant benets on lung function, respiratory symptom scores, and pulmonary exacerbation rates.2 Somewhat surprisingly and currently incompletely understood, there was also a signicant gain in body weight. e eect size of lung function improvement exceeded that seen in any previous study evaluating a therapeutic intervention in CF and was certainly bigger than most people in the eld had expected. ese results have clearly demonstrated that CFTR-directed therapy can be highly eective and appear to have catalyzed a resurgence of enthusiasm among pharmaceutical companies developing small molecule therapies.
