23 February 2024 - If approved, Kalydeco will be the first and only medicine approved in Europe to treat the underlying cause of cystic fibrosis in babies as young as 1 month with specific mutations in the CFTR gene.

Vertex Pharmaceuticals today announced that the EMA’s CHMP adopted a positive opinion for the label expansion of Kalydeco (ivacaftor) for the treatment of infants with cystic fibrosis ages 1 month to less than 4 months old who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator gene: R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

Read Vertex Pharmaceuticals press release