Kalydeco ivacaftor regulatory update

FDA approved Kalydeco ivacaftor to treat cystic fibrosis in patients aged 6 and older who have at least 1 copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. FDA said Kalydeco is the first drug approved that targets the defective CFTR protein, which is the underlying cause of CF. About 4% of U.S. CF patients, or about 1,200 people, have the specific G551D mutation. The approval comes just over 3 months after Vertex submitted an NDA for Kalydeco in October and is well ahead of its April 28 PDUFA date. It has Orphan Drug status in the U.S.

Vertex launched the small molecule CFTR potentiator at an annual wholesale acquisition cost of $294,000. Vertex will provide the drug for free to uninsured patients with an annual household income of $150,000 or less. The company will also offer a co-pay assistance program for certain patients with commercial insurance. ...