Cystic fibrosis (CF), combination treatment with ivacaftor/tezacaftor/elexacaftor in patients ≥ 12 years (heterozygous for F508del and RF mutation)
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-688
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/elaxacaftor in people aged 12 years and over with cystic fibrosis who are heterozygous for the F508del mutation in the CFTRgene and carry a residual-function mutation on the second allele, the pharmaceutical manufacturer submitted the Phase III, multicentre, randomised, double-blind, controlled trial VX18-445-104 (hereinafter ‘Study 104’) was submitted by the pharmaceutical company.
Individuals aged 12 years and over 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.
Consequently, the pharmaceutical manufacturer did not submit any study for this patient population 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, additional benefit is not proven for people aged 12 years and over 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 people aged 12 years and over 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.
