5 July 2023 - Nearly 300 children with cystic fibrosis and two copies of the F508del mutation are now eligible for the first time for a medicine that can treat the underlying cause of their disease.

Vertex Pharmaceuticals today announced that the European Commission has granted approval for the label extension of Orkambi (lumacaftor/ivacaftor) for the treatment of children with cystic fibrosis ages 1 to <2 years old who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene, the most common form of the disease.

Read Vertex Pharmaceuticals press release