NICE rebuffs Vertex's Orkambi

The U.K.'s NICE issued draft guidance recommending against the use of Orkambi ivacaftor/lumacaftor to treat cystic fibrosis in patients homozygous for the F508 CF transmembrane conductance regulator ( CFTR) mutation, saying the treatment "could not be considered a cost effective use of NHS resources." Orkambi, which NICE said costs L104,000 ($149,614) annually, is marketed by Vertex Pharmaceuticals Inc. (NASDAQ:VRTX).

NICE said Orkambi added a "modest" benefit compared to standard of care, and noted that most patients receiving Orkambi remain on SOC. NICE said England has about 2,750 CF patients eligible for Orkambi. ...