Vertex Pharmaceuticals has announced data from a long-term follow-up study (PERSIST) which shows significant improvements in lung function, respiratory symptoms and weight gain among people treated with Kalydeco. The study enrolled people with cystic fibrosis (CF) with at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Kalydeco is the first medicine to treat the underlying cause of CF, a rare, genetic disease caused by defective or missing CFTR proteins resulting from mutations in the CFTR gene. In people with the G551D mutation, Kalydeco helps the defective CFTR protein function more normally.
A total of 74 adults and adolescents (ages 12 or older), who were first treated with Kalydeco in an earlier study (STRIVE), continued treatment in the PERSIST study. A 9.5 percentage-point mean absolute improvement from the STRIVE baseline in lung function was observed at week 96. Twenty-five children (ages 6 to 11) who were first treated with Kalydeco in the ENVISION study, also continued treatment in PERSIST. A 10.1 percentage-point mean absolute improvement from the ENVISION baseline in lung function was observed at week 72.
“Cystic fibrosis is a progressive disease and as patients get older, lung damage progresses and often becomes irreversible,” said Kalydeco investigator, Jane Davies, Royal Brompton Hospital and Imperial College, London. “The goal of this study was to start to help us understand whether patients might benefit from treatment with Kalydeco before they show severe signs and symptoms of CF. The initial findings are encouraging and support longer-term studies and evaluation.”
