Cystic fibrosis (CF), F508del mutation, heterozygous and RF 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-796
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
To assess 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 residual-function mutation on the second allele, the pharmaceutical manufacturer did not submit any direct comparative studies against the appropriate comparator therapy.
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 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 residual-function mutation on the second allele
An additional benefit is not proven.
Overall, additional benefit is not proven for children aged 6 to 11 with cystic fibrosis who are heterozygous for the F508del mutation and carry a residual-function mutation on the second allele.
Overall assessment
On balance, additional benefit is not proven for children aged 6 to 11 with cystic fibrosis who are heterozygous for the F508del mutation and carry a residual-function mutation on the second allele.
Courtesy translation only, please refer to the German original.
