Ivacaftor (17) – Kalydeco®
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) |
| ICD-10 codes (AIS) | E84.0Cystic fibrosis with pulmonary manifestations, E84.1Cystic fibrosis with intestinal manifestations, E84.80, E84.87, E84.88, E84.9Cystic fibrosis, unspecified |
| Alpha-ID codes (AIS) | I129376Neonatal hepatobiliary disease in cystic fibrosis, I130516Cystic fibrosis with other multiple manifestations, I18531Cystic fibrosis, I2487Cystic fibrosis with pulmonary manifestation, I2488Cystic fibrosis with intestinal manifestation, I32495Cystic fibrosis with manifestations in the lungs and digestive tract |
| ORPHAcodes (AIS) | 586Neonatal hepatobiliary disease in cystic fibrosis, 586Cystic fibrosis with other multiple manifestations, 586Cystic fibrosis, 586Cystic fibrosis with pulmonary manifestation, 586Cystic fibrosis with intestinal manifestation, 586Cystic fibrosis with manifestations in the lungs and digestive tract |
| DDD | 0.15 g O |
| Therapeutic area | Metabolic diseases Cystic fibrosis (CF) Orphan (turnover limit) |
| Reason for procedure | New therapeutic indication – Orphan turnover exceeded |
| Specialty | Bundling |
| Therapeutic indication of the resolution |
|---|
|
Treatment of infants with cystic fibrosis (CF) aged 4 - < 6 months who have an R117H CFTR mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. |
| Subpopulation | Indication | Comparator |
|---|---|---|
| Infants with cystic fibrosis aged 4 to < 6 months who have an R117H mutation in the CFTR gene. | Best-Supportive-Care |
Studies and Results
|
No. of studies
(best subpopulation) |
1 (VX15-770- 124) |
|---|---|
|
Study design
(best subpopulation) |
Evidence transfer |
|
Meta analysis
(best subpopulation) |
no |
- 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.
Associated procedures
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