Data from a Phase III trial of Vertex Pharmaceuticals' Kalydeco (ivacaftor) has been published ion the New England Journal of Medicine.
Kalydeco targets the defective protein responsible for cystic fibrosis.
The STRIVE study evaluated 161 patients aged 12 or older who received at least one dose of either Kalydeco as a single 150mg tablet or placebo twice daily and showed improvements in lung function and other measures of disease throughout the 48-week study.
The study reported a 10.6% mean absolute improvement and 16.7% relative mean improvement in lung function from baseline compared to placebo through to week 24.
Through to week 48, the mean absolute improvement in lung function was 10.5% and the relative mean improvement was 16.9% from baseline compared to placebo.
The company has submitted a new drug application to the US Food and Drug Administration and a marketing authorisation application to the European Medicines Agency.