Ivacaftor / Tezacaftor / Elexacaftor (14) – Kaftrio®
Cystic fibrosis, F508del mutation, heterozygous and RF mutation, ≥ 2 to ≤ 5 years, combination with ivacaftor
Characteristics
| Start date | 01.12.2023 – Marketing authorisation: 22.11.2023 |
|---|---|
| Resolution | 16.05.2024 |
| INN | Ivacaftor/Tezacaftor/Elexacaftor |
| Brand name | Kaftrio® |
| Pharm. company | Vertex Pharmaceuticals (Germany) GmbH |
| G-BA Procedure ID | D-1020 |
| ATC code | R07AX32 Other respiratory system products (R07AX) |
| Therapeutic area | Metabolic diseases Orphan (turnover limit) |
| Reason for procedure | New therapeutic indication – Orphan turnover exceeded |
| Specialty | Bundling |
Studies and Results
- Clinical trials
- The pharmaceutical manufacturer has submitted data for the assessment of the additional benefit of ivacaftor/tezacaftor/Elexacaftor in combination with Ivacaftor for children aged 2 to ≤ 5 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a residual-function mutation on the second allele, the pharmaceutical manufacturer has not provided any direct comparative studies against the appropriate comparator therapy.
Children aged 2 to ≤ 5 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a residual-function mutation on the second allele
- In combination with ivacaftor, an additional benefit is not proven in children aged 2 to ≤ 5 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a residual-function mutation on the second allele.
- For the assessment of the additional benefit of ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor in children aged 2 to ≤ 5 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a function-preserving mutation on the second allele, the pharmaceutical manufacturer has not submitted any direct comparative studies against the appropriate comparator therapy.
- Based on the information provided by the pharmaceutical manufacturer, it is not possible to extrapolate the additional benefit to patients with a different mutation type or to older patients within the population for the therapeutic indication.
- Overall, an additional benefit is not proven for children aged 2 to ≤ 5 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a residual-function mutation on the second allele.
- Overall assessment
- Overall, additional benefit is not proven for children aged 2 to ≤ 5 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a residual-function mutation on the second allele.
Courtesy translation only, please refer to the German original.
Associated procedures
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