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 |
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| 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) |
| 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 |
| 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 |
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Ivacaftor/tezacaftor/lexacaftor is used in combination with ivacaftor for the treatment of cystic fibrosis in pediatric patients aged 2 to ≤ 5 years who are heterozygous for the F508del mutation in the CFTR gene and have a mutation on the second allele that is not a minimal function, gating (including R117H) or residual function mutation or where the mutation on the second allele is unknown (other mutations). |
| Subpopulation | Indication | Comparator |
|---|---|---|
| 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 not a minimal function, gating (incl. R117H) or residual function mutation or in whom the mutation on the second allele is unknown (other mutations) | Best supportive care |
Studies and Results
|
No. of studies
(best subpopulation) |
0 (Data not accepted) |
|---|---|
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Study design
(best subpopulation) |
Data not accepted (Dossier: Evidence transfer) |
- 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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