Cystic fibrosis, F508del mutation, heterozygous and gating mutation, ≥ 6 to ≤ 11 years, combination with ivacaftor)
Characteristics
Start date
15.02.2022
–
Marketing authorisation:
07.01.2022
Resolution
04.08.2022
INN
Ivacaftor/Tezacaftor/Elexacaftor
Brand name
Kaftrio®
Pharm. company
Vertex Pharmaceuticals (Germany) GmbH
G-BA Procedure ID
D-775
ATC code
R07AX32
Other respiratory system products (R07AX)
DDD
2
O
Therapeutic area
Metabolic diseases
Orphan (turnover limit)
Reason for procedure
New therapeutic indication
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 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
For the assessment of the additional benefit of ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor 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.
It is not possible to extrapolate the additional benefit to the population in the present therapeutic indication, based on the information provided by the pharmaceutical manufacturer, either to patients with a different mutation type or to older patients.
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 therefore not proven.
Courtesy translation only, please refer to the German original.
