Vertex’s Kaftrio + Ivacaftor Receive the EC’s Approval to Treat Cystic Fibrosis in People Aged 12 Years and Older

August 24, 2020 Auto Bot approval, Cystic fibrosis, EC, Receive, Uncategorized, Vertex 0

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

Click here ­to­ read full press release/ article | Ref: PRNewswire | Image: Bloomberg