Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are small molecules aimed at improving CFTR function by specifically targeting the different classes of CFTR mutations. Recent Phase II studies of triple therapy, including new generation correctors, have demonstrated spectacular improvements in forced expiratory volume in 1s (FEV1), likely to translate into improved quality of life and increased life expectancy. Within the next 5 years, a highly effective CFTR modulator therapy will probably be approved for most cystic fibrosis patients, including those carrying at least one copy of the F508del mutation (88% of Belgian patients). Patients with well-preserved lungs will benefit most from these treatments.
Cystic fibrosis, cystic fibrosis transmembrane conductance regulator (CFTR), modulators, VX-445, VX-659
What is already known about the topic?
CFTR modulators are currently the privileged research path towards a more fundamental treatment of cystic fibrosis.
What does this article bring up for us?
Highly effective combinations of CFTR modulators, including new generation correctors, will probably become available within the next 5 years and benefit to most Belgian patients with cystic fibrosis.