Ivacaftor (Kalydeco, Vertex Pharmaceuticals) is already FDA approved to treat cystic fibrosis in patients aged 6 months and older. The new approval was based on data from ARRIVAL, a phase 3, open-label, safety cohort study of six children with cystic fibrosis aged 4 months to less than 6 months. All children had at least one of 10 mutations in the CFTR gene. In this cohort, treatment with ivacaftor demonstrated a similar safety profile to older children and adults. In Europe the Orphan Designation for Ivacaftor was granted on 08 July 2008 (EC Decision No. EU/3/08/556) and on 28 September 2011 the CHMP accepted the Applicant’s request for accelerated assessment. On 23 July 2012 Ivacaftor obtained the marketing authorisation valid throughout the European Union and currently it is still used on its own to treat cystic fibrosis only in patients aged 6 months and above who have one of the following mutations (changes) in the gene for a protein called ‘cystic fibrosis transmembrane conductance regulator’ (CFTR): R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R.
