Cystic fibrosis (CF), F508del mutation, heterozygous and gating mutation, ≥ 6 to ≤ 11 years, combination with ivacaftor / tezacaftor / elexacaftor).
Characteristics
Start date
15.02.2022
–
Marketing authorisation:
07.01.2022
Resolution
04.08.2022
INN
Ivacaftor
Brand name
Kalydeco®
Pharm. company
Vertex Pharmaceuticals (Germany) GmbH
G-BA Procedure ID
D-795
ATC code
R07AX02
Other respiratory system products (R07AX)
DDD
0.3
g
O
Therapeutic area
Metabolic diseases
Orphan (turnover limit)
Reason for procedure
New therapeutic indication
–
Orphan turnover exceeded
Specialty
Bundling
Studies and Results
Clinical trials
The key study, VX18-445-104, which the pharmaceutical manufacturer cites in its argument for extending the additional benefit to patients aged 12 and over, was already deemed unsuitable in the context of the benefit assessments for this age group due to the study duration being insufficient.
Children aged 6 to 11 with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a gating mutation (including R117H) on the second allele
An additional benefit is not proven.
For the assessment of the additional benefit of ivacaftor in combination with ivacaftor/tezacaftor/elexacaftor in children aged 6 to 11 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a gating mutation (including R117H) on the second allele, the pharmaceutical manufacturer has not submitted any direct comparative studies against the appropriate comparator therapy.
Based on the information provided by the pharmaceutical manufacturer, it is not possible to extrapolate the additional benefit to patients with a different mutation type or to older patients within the population for the therapeutic indication.
Overall, for children aged 6 to 11 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a gating mutation (including R117H) on the second allele, additional benefit is not proven.
Overall assessment
On balance, for children aged 6 to 11 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a gating mutation (including R117H) on the second allele, additional benefit is not proven.
Courtesy translation only, please refer to the German original.
