Cystic fibrosis (CF), combination treatment with ivacaftor/tezacaftor/elexacaftor in patients ≥ 12 years (heterozygous for F508del and gating mutation (incl. R117H))
Characteristics
Start date
01.06.2021
–
Marketing authorisation:
26.04.2021
Resolution
19.11.2021
INN
Ivacaftor
Brand name
Kalydeco®
Pharm. company
Vertex Pharmaceuticals (Germany) GmbH
G-BA Procedure ID
D-686
ATC code
R07AX02
Other respiratory system products (R07AX)
DDD
0.15
mg
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 people aged 12 years and over with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a gatingmutation (including R117H) on the second allele, the pharmaceutical manufacturer submitted the Phase III VX18-445-104 (hereinafter ‘Study 104’) was submitted by the pharmaceutical company.
People aged 12 years and over 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 additional benefit is not proven.
Consequently, the pharmaceutical manufacturer did not submit any study for the relevant patient population in question that would have been suitable for assessing the additional benefit of ivacaftor in combination with ivacaftor/tezacaftor/elexacaftor compared with the appropriate comparator therapy.
Overall assessment
Overall, additional benefit is not proven for individuals aged 12 years and over who are heterozygous for the F508del mutation in the CFTR gene and carry a gating mutation (including R117H) on the second allele.
Courtesy translation only, please refer to the German original.
