EC approves Vertex' Kaftrio and ivacaftor combination

The European Commission has approved Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for cystic fibrosis (CF) patients aged 12 and older with one F508del mutation and one minimal function mutation (F/MF), or two F508del mutations (F/F) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

This means that, for the first time, up to 10,000 people in Europe with CF who have one F508del mutation and one minimal function mutation, will be eligible for a CFTR modulator that treats the underlying cause of disease.

Approval of the Vertex Pharmaceuticals (Nasdaq: VRTX) triple combination regimen also expands the number of treatment options available to people with CF who have two copies of the F508del mutation, the most common CF causing mutation worldwide.