Ivacaftor / Tezacaftor / Elexacaftor (17) – Kaftrio®

• Clinical trials
  • The VX21-445-124 trial is a randomised controlled trial comparing ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor versus placebo, in each case in addition to standard of care.

Adults, adolescents and children aged 2 years and over with cystic fibrosis who have at least one non-Class I mutation, which is a gating mutation and is not an F508del mutation, in the CFTR gene

• For adults, adolescents and children aged 2 years and over with cystic fibrosis who have at least one non-Class I mutation in the CFTR gene that is a gating mutation and is not an F508del mutation, an additional benefit is not proven.
• An additional benefit is not proven.
• There are no direct comparative data available for ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor against the appropriate comparator therapy, ivacaftor.
• The study VX21-445-124, primarily cited by the pharmaceutical manufacturer, is not suitable for establishing additional benefit.
• The study is a randomised controlled trial comparing ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor against placebo, in each case in addition to standard of care. No comparison was made against the appropriate comparator therapy, ivacaftor, as determined by the G-BA.
• The single-arm observational studies cited in support by the pharmaceutical manufacturer (VX22-CFD-016, HEOR-23-445-014, Burgel 2024, Cromwell 2024) and the extension study VX21-445-125 also do not allow for a comparison with the appropriate comparator therapy specified by the G-BA.
• Consequently, the data submitted by the pharmaceutical manufacturer are not suitable for drawing conclusions regarding the additional benefit of ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor compared with the appropriate comparator therapy.

Courtesy translation only, please refer to the German original.