Ivacaftor (27) – Kalydeco®

• Clinical trials
  • To assess the additional benefit of ivacaftor in combination with ivacaftor/tezacaftor/elexacaftor for the treatment of children aged 6 to 11 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a mutation on the second allele that is neither a minimal-function, nor a gating (including R117H) and no residual function mutation, or in whom the mutation on the second allele is unknown (other mutations), the pharmaceutical manufacturer has not submitted any study data.

Children aged 6 to 11 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a mutation on the second allele that is neither a minimal function, nor a gating (including R117H)or residual function mutation, or in whom the mutation on the second allele is unknown (other mutations)

• An additional benefit is not proven.
• Overall, the pharmaceutical manufacturer did not submit any study for the present therapeutic indication that would have been suitable for assessing the additional benefit of ivacaftor in combination with ivacaftor/tezacaftor/elexacaftor compared with the appropriate comparator therapy.
• An additional benefit of ivacaftor in combination with ivacaftor/tezacaftor/elexacaftor compared with the appropriate comparator therapy is not proven.
• Overall assessment
  • Taking into account the available evidence on the medical benefit of ivacaftor in combination with ivacaftor/tezacaftor/Elexacaftor, the progressive course of the disease and the statements from medical societies regarding the current reality of care, Ivacaftor in combination with Ivacaftor/Tezacaftor/Elexacaftor may represent a relevant treatment option for individual patients aged 6 to 11 years who are heterozygous for the F508del mutation in the CFTR gene and carry an unknown or other mutation on the second allele.

Courtesy translation only, please refer to the German original.