EMA — authorised 23 July 2012
- Application: EMEA/H/C/002494
- Marketing authorisation holder: Vertex Pharmaceuticals (Ireland) Limited
- Local brand name: Kalydeco
- Indication: Kalydeco tablets are indicated: - As monotherapy for the treatment of adults, adolescents, and children aged 6 years and older and weighing 25 kg or more with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (Class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. - In a combination regimen with tezacaftor/ivacaftor tablets for the treatment of adults, adolescents, and children aged 6 years and older with cystic fibrosis (CF) who are homoz
- Pathway: accelerated assessment
- Status: approved