Kalydeco ivacaftor regulatory update

FDA approved an expanded label for Kalydeco ivacaftor to treat cystic fibrosis in patients who have 1 of 10 mutations in the CF transmembrane conductance regulator ( CFTR) gene to include patients ages 2-5. The drug was indicated in patients ages <=6. The 10 mutations are G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D and R117H. A weight-based oral granule formulation of Kalydeco is available for patients ages 2-5. Vertex said there are about 300 patients in the U.S. in the age group who have 1 of the 10 mutations. ...