Cystic fibrosis (CF), combination therapy with tezacaftor/ivacaftor in patients 6 to < 12 years (homozygous for F508del mutation)
Characteristics
Start date
01.12.2020
–
Marketing authorisation:
25.11.2020
Resolution
20.05.2021
INN
Ivacaftor
Brand name
Kalydeco®
Pharm. company
Vertex Pharmaceuticals (Ireland) Limited
G-BA Procedure ID
D-623
ATC code
R07AX02
Other respiratory system products (R07AX)
DDD
0.15
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 demonstrate the additional benefit of IVA + TEZ/IVA in children aged 6 to < 12 years with cystic fibrosis who are homozygous for the F508del mutation, the pharmaceutical manufacturer submitted the single-arm study VX13-661-113 (hereinafter referred to as Study 113) was submitted by the pharmaceutical company to demonstrate the added benefit of IVA + TEZ/IVA.
In addition, the pharmaceutical manufacturer submitted the results of the 8-week RCT VX16-661-115 (IVA + TEZ/IVA vs. placebo; hereinafter referred to as Study 115) and the single-arm extension study VX17-661-116 (hereinafter referred to as Study 116).
Children with cystic fibrosis aged 6 to < 12 years who are homozygous for the F508del mutation
For children with cystic fibrosis aged 6 to < 12 years who are homozygous for the F508del mutation, the additional benefit of ivacaftor in combination with tezacaftor/ivacaftor compared with the appropriate comparator therapy 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 IVA + TEZ/IVA compared with the appropriate comparator therapy.
Overall, additional benefit is not proven for patients aged 6 years and over but under 12 years with cystic fibrosis who are homozygous for the F508del mutation in the CFTR gene.
Overall assessment
On balance, additional benefit is not proven for patients aged 6 years or older but under 12 years with cystic fibrosis who are homozygous for the F508del mutation in the CFTR gene.
Courtesy translation only, please refer to the German original.
