Ivacaftor (19) – Kalydeco®

• Clinical trials
  • In addition, the pharmaceutical manufacturer submitted the results of the 8-week RCT VX16-661-115 (IVA + TEZ/IVA vs. IVA; hereinafter referred to as Study 115) and the single-arm extension study VX17-661-116 (hereinafter referred to as Study 116).

Children with cystic fibrosis aged 6 to < 12 years who are heterozygous for the F508del mutation and carry one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272–26A→G, and 3849+10kbC→T

• For children with cystic fibrosis aged 6 to < 12 years who are heterozygous for the F508del mutation and carry one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272–26A→G, and 3849+10kbC→T, the additional benefit of tezacaftor/ivacaftor in combination with ivacaftor compared with the appropriate comparator therapy is not proven.
• In its dossier for the assessment of the additional benefit of IVA + TEZ/IVA, the pharmaceutical manufacturer does not present any direct comparative studies against the appropriate comparator therapy, Best Supportive Care (BSC).
• Furthermore, no indirect comparisons were submitted to address the issue of benefit assessment.
• Consequently, the pharmaceutical manufacturer has not submitted any study for this patient population that would have been suitable for assessing the additional benefit of IVA + TEZ/IVA compared with the appropriate comparator therapy.

Courtesy translation only, please refer to the German original.