Cystic fibrosis (CF), patients from 4 to < 6 months, R117H mutation
Characteristics
Start date
01.12.2020
–
Marketing authorisation:
03.11.2020
Resolution
20.05.2021
INN
Ivacaftor
Brand name
Kalydeco®
Pharm. company
Vertex Pharmaceuticals (Ireland) Limited
G-BA Procedure ID
D-619
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
In the single-arm, open-label Phase III registration trial VX15-770-124, the relevant cohort comprised 7 infants, totalling 6 infants aged 4 to < 6 months.
Infants with cystic fibrosis aged 4 to < 6 months who carry an R117H mutation in the CFTR gene
Hint for a non-quantifiable additional benefit
Due to the uncertainty arising from the extrapolation of the additional benefit to a younger population, there is a hint of a non-quantifiable additional benefit.
Due to the uncertainty surrounding the transfer of the additional benefit to a younger population, a hint is derived overall.
Overall assessment / Conclusion
On balance, the G-BA concludes that the transferability of the additional benefit of ivacaftor from patients aged 18 years and over to infants with cystic fibrosis aged 4 to < 6 months who carry an R117H mutation in the CFTR gene, is assumed – particularly given the comparable clinical presentation, the progressive course of the disease and the limitations in conducting clinical trials in this age group, whilst taking into account the safety data for children with gating mutations.
However, the additional benefit is non-quantifiable, as the current scientific evidence does not permit this at this stage.
Courtesy translation only, please refer to the German original.
