Vanzacaftor / tezacaftor / deutivacaftor for the treatment of cystic fibrosis

Vanzacaftor/tezacaftor/deutivacaftor is in development for the treatment of cystic fibrosis in patients with at least one F508del mutation (faulty gene). Cystic fibrosis is a rare, chronic, and life-shortening genetic disease caused by a faulty gene that affects the movement of salt and water in and out of cells. It is 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 build-up 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 cystic fibrosis. It is an inherited condition, and the symptoms include recurring chest infections, wheezing, coughing, shortness of breath, damage to the airways and jaundice. Current treatment options have limited efficacy in some patients with cystic fibrosis.