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) |
| 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 |
|---|
|
Ivacaftor/tezacaftor/lexacaftor is used as a combination treatment 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 carry a residual function mutation on the second allele. |
| 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 residual function mutation on the second allele | Best supportive care |
Studies and Results
|
No. of studies
(best subpopulation) |
0 (Data not accepted) |
|---|---|
|
Study design
(best subpopulation) |
Data not accepted (Dossier: Evidence transfer) |
- 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
<< List of all resolutions