Posted by Michael Wonder on 15 Sep 2020
Vertex announces European Medicines Agency type II variation marketing authorisation application validation for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor in people with one copy of the F508del mutation
14 September 2020 - Vertex Pharmaceuticals today announced the EMA has validated a type II variation marketing authorisation application for the expanded indication of Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor to treat cystic fibrosis in patients ages 12 years and older with at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene.
If approved, eligible patients who have one copy of the F508del mutation and another cystic fibrosis transmembrane conductance regulator mutation, such as a gating or residual function mutation, will also be eligible for treatment.
The application is supported by positive results from the global Phase 3 trial (445-104) with Kaftrio announced in July 2020.
