Vertex Pharmaceuticals announced the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen. The submission was supported by previously disclosed positive results of two global Phase 3 studies in people with cystic fibrosis (CF): a 24-week study in people with one F508del mutation and one minimal function mutation and a 4-week study in people with two F508del mutations. Both Phase 3 studies showed statistically significant improvements in lung function (percent predicted forced expiratory volume in one second; ppFEV1), which was the primary endpoint, and in all key secondary endpoints. In these studies, the triple combination regimen was generally well tolerated.
“Today marks a significant milestone towards our efforts to bring new medicines to more people around the world who are living with cystic fibrosis,” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We are looking forward to working with the EMA on this important application.”
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Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.