The opportunity to redefine the treatment of major diseases is what drives everyone at Vertex. Our development portfolio represents the fruit of these efforts. Spend some time here and look around. Click on the pipeline chart to get information on our development programs and links to selected published articles. Go further and see how, just maybe, disease challenges may be overcome with innovation.

VX-770
(Cystic Fibrosis)

Cystic Fibrosis (CF) affects about 30,000 people in the United States and approximately 70,000 people worldwide. Cystic fibrosis is caused by genetic mutations that result in a malfunctioning or missing Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) proteins on cell surfaces. These missing or malfunctioning CFTR proteins result in an imbalance of salt and water in the airways, causing a cascade of mucus plugging, infection and inflammation that characterizes CF. This cascade accounts for a large portion of the morbidity and mortality seen with CF.

VX-770, an investigational CFTR potentiator, aims to increase chloride ion transport through the defective CFTR protein. VX-770 was advanced into preclinical development based on successful collaborative research with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) that incorporated capabilities and proprietary ion channel research from Vertex's San Diego research site.

Three trials of VX-770 are fully enrolled as part of a global Phase 3 registration program focused on patients with the G551D mutation. These trials include the Phase 3 STRIVE trial in patients aged 12 years and older with the G551D mutation, the Phase 3 ENVISION trial in patients aged six to 11 years with the G551D mutation, and the Phase 2 DISCOVER trial in patients aged 12 and older homozygous for the F508del mutation.

Data from the Phase 3 registration program of VX-770 are expected in the first half of 2011, and Vertex expects to submit a New Drug Application to the U.S. Food and Drug Administration in the second half of 2011.