Orkambi ivacaftor/lumacaftor regulatory update

FDA approved an NDA for Orkambi ivacaftor/lumacaftor from Vertex to treat cystic fibrosis in patients ages >=12 who are homozygous for the F508del mutation of the CF transmembrane conductance regulator ( CFTR) gene. On July 2, Vertex said it would 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 (see BioCentury, May 18). The therapy combines lumacaftor ( VX-809), a small molecule CFTR corrector, and Kalydeco ivacaftor, a small molecule potentiator of CFTR. ...