Vertex Pharmaceuticals products

We’re investigating a portfolio of other small molecule medicines aimed at treating the underlying cause of CF. This includes CFTR potentiators, which are designed to keep CFTR proteins at the cell surface open longer to improve the flow of salt and water across cell membranes, helping to hydrate and clear mucus from the airways. We’re also researching additional small molecules to address the trafficking and processing defect of the CFTR protein to enable it to move through cells and reach the surface.

We’re investigating other potential small molecule medicines aimed at treating the underlying cause of CF. VX-561 (deutivacaftor) is an investigational once daily potentiator designed to keep CFTR proteins at the cell surface open longer to improve the flow of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

Vertex is focused on developing transformative medicines for people with serious and life-threatening diseases. To do this, we conduct clinical trials to assess the safety and effectiveness of investigational medicines, which, if established, will help us obtain approvals from regulatory authorities. These approvals, in turn, are required before medicines can be made widely available to patients.

Cystic fibrosis (CF) is a rare, chronic and life-shortening genetic disease. It’s a progressive, multi-system disease that affects the lungs, liver, gastrointestinal tract, pancreas, sinuses, sweat glands and reproductive tract. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and inflammation, resulting in progressive lung damage and premature death in many people with CF. Other complications can include malabsorption and failure to thrive due to pancreatic disease, diabetes, liver disease, bone disease or osteoporosis, depression and anxiety. The median age of death is in the early 30s.