Vertex Pharma Reports Ivacaftor Study Results - Quick Facts

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) announced the results of a two-part proof-of-concept study of ivacaftor in 24 people with cystic fibrosis who have a residual function mutation. In part one of the study, a statistically significant improvement in mean absolute lung function was observed after treatment with ivacaftor for two weeks compared with placebo. In part two of the study, improvements in lung function were observed after eight weeks of treatment, the company said. In the study, ivacaftor was generally well-tolerated.

Based on the data, Vertex plans to initiate a larger Phase 3 study in people with residual function mutations that will evaluate longer-duration treatment with ivacaftor.