Ivacaftor / Tezacaftor / Elexacaftor (15) – Kaftrio®
Cystic fibrosis, F508del mutation, heterozygous and other or unknown 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-1021 |
| 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
- To assess 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 F508delmutation in the CFTR gene and who carry a mutation on the second allele that is neither a minimal function, a gating (including R117H) nor a residual function mutation, or in whom the mutation on the second allele is unknown (other mutations), no directly comparative studies against the appropriate comparator therapy have been submitted by the pharmaceutical manufacturer.
Children aged 2 to ≤ 5 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a mutation on the second allele that is neither a minimal function mutation, nor a gating mutation (including R117H)- or residual function mutation, or in whom the mutation on the second allele is unknown (other mutations)
- In combination with ivacaftor, no additional benefit has been demonstrated in children aged 2 to ≤ 5 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a mutation on the second allele that is neither a minimal function, nor a gating (including R117H) or residual function mutation on the second allele, or in whom the mutation on the second allele is unknown (other mutations), additional benefit is not proven.
- An additional benefit of ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor compared with the appropriate comparator therapy is not proven in this therapeutic indication.
- Overall assessment
- In the overall assessment, for children aged 2 to ≤ 5 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a mutation on the second allele that is neither a minimal-function, nor a gating (including R117H) or residual function mutation, or in whom the mutation on the second allele is unknown (other mutations), additional benefit is not proven.
Courtesy translation only, please refer to the German original.
Associated procedures
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