Posted by Michael Wonder on 03 Mar 2025
Vertex receives CHMP positive opinion for expanded label for Kaftrio in combination with ivacaftor for people with cystic fibrosis to include rare mutations
28 February 2025 - If approved, approximately 4,000 people with cystic fibrosis in the European Union will be eligible for a medicine that treats the underlying cause of their disease for the first time.
Vertex Pharmaceuticals today announced that the EMA's CHMP adopted a positive opinion for the label expansion of Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor for the treatment of people with cystic fibrosis aged 2 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator gene.
