CHMP grants positive opinion for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination With Kalydeco (ivacaftor) in people ages 12 and older With Cystic Fibrosis with the most common genotypes.- Vertex

Vertex Pharmaceuticals Incorporated announced that the ?European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for Kaftrio -(ivacaftor/tezacaftor/elexacaftor) in a combination regimen with Kalydeco (ivacaftor) 150 mg to treat people with cystic fibrosis (CF) ages 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.

If approved, up to 10,000 people in Europe with CF ages 12 and older who have one F508del mutation and one minimal function mutation would be newly eligible for a medicine that targets the disease- causing protein defect. Additionally, people 12 years of age and older who have two F508del mutations and who are currently eligible for one of Vertex’s other EMA-approved cystic fibrosis medicines would be eligible for the new triple combination regimen.

The CHMP positive opinion was based on the results of two global Phase III studies in people with CF: a 24-week placebo-controlled study in people ages 12 years and older with one F508del mutation and one minimal function mutation and a 4-week head-to-head study of the triple combination therapy in comparison to tezacaftor/ivacaftor in people ages 12 years and older with two F508del mutations. Both Phase III studies showed statistically significant and clinically meaningful improvements in lung function (percent predicted forced expiratory volume in one second; ppFEV1), which was the primary endpoint, and in all key secondary endpoints. In both studies, the ivacaftor/tezacaftor/elexacaftor plus ivacaftor combination regimen was generally well tolerated.