Shots:

• The EC has granted MAA to Kaftrio (ivacaftor/tezacaftor/lumacaftor) + ivacaftor (150mg) to treat people with CF aged ≥12yrs. with one F508del mutation and one minimal function mutation (F/MF)- or two F508del mutations (F/F) in the CFTR gene
• The MAA is based on two P-III studies i.e. a 24wks. study in 403 people with one F508del mutation and one F/MF & a 4wks. study in 107 people with two F508del mutations (F/F). The studies demonstrated improvements in lung function (1EPs) and all 2EPs and were generally well-tolerated in both studies
• Kaftrio is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface

­ Ref: PRNewswire | Image: Vertex