FDA approves Vertex's CF drug

FDA approved Kalydeco ivacaftor from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) to treat cystic fibrosis in patients aged six and older who have at least one 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 three months after Vertex submitted an NDA for Kalydeco in October and is well ahead of its April 28 PDUFA date.

Vertex plans to launch the small molecule CFTR potentiator this week with 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. ...