VX-522 for treatment of CFTR genotype cystic fibrosis unresponsive to CFTR modulator treatment

VX-522 is in development for treatment of cystic fibrosis in patients with cystic fibrosis transmembrane conductance regulator (CFTR) genotype which are not responsive to CFTR modulator therapy. 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, it 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. It is an inherited condition, and symptoms include recurring chest infections, wheezing, coughing, shortness of breath, damage to the airways and jaundice. Current therapies are not effective in some patients with CFTR mutations, and only control symptoms rather than addressing the cause of the disease, meaning patients have limited therapeutic options available.