Cystic fibrosis (CF), F508del mutation, heterozygous and RF 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-776
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
To assess 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 residual-function mutation on the second allele, the pharmaceutical manufacturer did not submit any direct comparative studies against the appropriate comparator therapy.
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 of ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor compared with the appropriate comparator therapy is not proven in the present therapeutic indication.
Overall, additional benefit is not proven for children aged 6 to 11 years with cystic fibrosis who are heterozygous for the F508del mutation and carry a residual-function mutation on the second allele.
Appropriate comparator therapy
The appropriate comparator therapy was defined as follows: 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
Appropriate comparator therapy for ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor: tezacaftor/ivacaftor in combination with ivacaftor
Courtesy translation only, please refer to the German original.
