Vertex’s Ivacaftor Benefits Residual Function Mutation Cystic Fibrosis Patients in First-of-Kind Study
Vertex Pharmaceuticals‘ two-part proof-of-concept study evaluating ivacaftor (under the name KALYDECO) in 24 cystic fibrosis patients shows data consistent with previous preclinical observations. An eight-week open-label period resulted in improved lung function in patients, initiating plans for a future Phase 3 trial. Each of the 24…