Ivacaftor (7) – Kalydeco®

• Clinical trials
  • To assess the additional benefit of IVA + TEZ/IVA in patients aged 12 years and over with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and who have a mutation on the second allele with residual CFTR function (RF mutation) on the second allele, the pharmaceutical manufacturer submitted the Phase III multicentre, randomised, double-blind, placebo-controlled crossover study VX14-661-108 (hereinafter ‘Study 108’) was submitted by the pharmaceutical company.

Patients aged 12 years and over with cystic fibrosis 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 is not proven.
• Consequently, the pharmaceutical manufacturer did not submit 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.
• Overall assessment
  • Overall, for patients aged 12 years and over with cystic fibrosis 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 whom additional benefit is not proven for treatment with IVA + TEZ/IVA.

Courtesy translation only, please refer to the German original.