Orkambi ivacaftor/lumacaftor regulatory update

EMA’s CHMP recommended approval of Orkambi ivacaftor/lumacaftor from Vertex to treat cystic fibrosis in patients ages >=12 who are homozygous for the F508del mutation in the CF transmembrane conductance regulator ( CFTR) gene. Vertex markets Orkambi in the U.S. for the indication (see BioCentury, July 13). The therapy combines lumacaftor ( VX-809), a small molecule CFTR corrector, and Kalydeco ivacaftor, a small molecule potentiator of CFTR. ...