Abstract and Introduction
Abstract
On January 31, 2012, the Food and Drug Administration (FDA) approved ivacaftor (Kalydeco, Vertex Pharmaceuticals, Inc., formerly known as VX-770) for the treatment of cystic fibrosis (CF) in patients 6 years of age and older who have the G551D mutation in the CF transmembrane regulator (CFTR) gene. As the first drug to target the defective CFTR protein rather than treat symptoms, ivacaftor represents a significant breakthrough for patients with CF. This development also reflects the success of a long-term collaborative effort between the manufacturer, an established network of CF centers and academic research laboratories located throughout Europe, North America, and Australia, and Cystic Fibrosis Foundation Therapeutics, Inc., the drug development branch of the Cystic Fibrosis Foundation.