BioCentury
ARTICLE | Top Story

FDA approves Vertex's Orkambi for CF

July 3, 2015 1:09 AM UTC

FDA approved Orkambi ivacaftor/lumacaftor from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) to treat cystic fibrosis patients aged 12 and older homozygous for the F508 mutation of the CF transmembrane conductance regulator ( CFTR) gene. Chief Commercial Officer Stuart Arbuckle said on a conference call that Vertex will launch Orkambi "within days" at an annual wholesale acquisition cost (WAC) of $259,000.

In May, FDA's Pulmonary-Allergy Drugs Advisory Committee voted 12-1 to support approval of Orkambi. The therapy combines lumacaftor ( VX-809), a small molecule CFTR corrector, and Kalydeco ivacaftor, a small molecule potentiator of CFTR (see BioCentury Extra, May 12). ...