Cystic fibrosis, F508del mutation, heterozygous and gating mutation, ≥ 2 to ≤ 5 years, combination with ivacaftor
Characteristics
Start date
01.12.2023
–
Marketing authorisation:
22.11.2023
Resolution
16.05.2024
INN
Ivacaftor/Tezacaftor/Elexacaftor
Brand name
Kaftrio®
Pharm. company
Vertex Pharmaceuticals (Germany) GmbH
G-BA Procedure ID
D-1019
ATC code
R07AX32
Other respiratory system products (R07AX)
Therapeutic area
Metabolic diseases
Orphan (turnover limit)
Reason for procedure
New therapeutic indication
–
Orphan turnover exceeded
Specialty
Bundling
Studies and Results
Children aged 2 to ≤ 5 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
In combination with ivacaftor in children aged 2 to ≤ 5 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, an additional benefit is not proven.
For the assessment of the additional benefit of ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor in children aged 2 to ≤ 5 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 the population in the present therapeutic indication, neither for patients with a different mutation type nor for older patients.
Overall assessment
On balance, for children aged 2 to ≤ 5 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, an additional benefit is not proven.
Courtesy translation only, please refer to the German original.
