As published in WestSide Story, University of Alabama at Birmingham School of Medicine and Denver’s National Jewish Health are participants in two new studies focusing on a pair of triple-drug treatments that may represent the first effective treatment for most cystic fibrosis patients—a chronic and incurable condition that often turns fatal. 1 in 3,000 (primarily northern European) are afflicted with this genetic disorder that affects mostly the lungs but also can impact the pancreas, liver, kidneys and intestine. Based on ivacaftor—a drug approved in 2012 to treat cystic fibrosis—the primary research centers include University of Alabama and Denver National Jewish Health. is a drug used to treat cystic fibrosis in people with certain mutations in the cystic fibrosis transmembrane conductance regulator(CFTR) gene (primarily the G551D mutation), who account for 4–5% cases of cystic fibrosis. It is also included in a combination drug, lumacaftor/ivacaftor (
Ivacaftor was developed by Vertex Pharmaceuticals in conjunction with the Cystic Fibrosis Foundation and is the first drug that treats the underlying cause rather than the symptoms of the disease. It was approved by the FDA in January 2012. It is one of the most expensive drugs, costing over US$300,000 per year, which has led to criticism of the high cost. The combination drug was approved by the FDA in July 2015.
Steven Rowe, University of Alabama
Price Watch: Ivacaftor–$300,000 per year.