Ivacaftor / Tezacaftor / Elexacaftor (9) – Kaftrio®
Cystic fibrosis (CF), F508del mutation, heterozygous and RF mutation, ≥ 6 to ≤ 11 years, combination with ivacaftor
Characteristics
| Start date | 15.02.2022 – Marketing authorisation: 07.01.2022 |
|---|---|
| Resolution | 04.08.2022 |
| INN | Ivacaftor/Tezacaftor/Elexacaftor |
| Brand name | Kaftrio® |
| Pharm. company | Vertex Pharmaceuticals (Germany) GmbH |
| G-BA Procedure ID | D-776 |
| 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 |
| DDD | 2 O |
| Therapeutic area | Metabolic diseases Cystic fibrosis (CF) Orphan (turnover limit) |
| Reason for procedure | New therapeutic indication |
| Specialty | Bundling |
| Therapeutic indication of the resolution |
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Kaftrio is indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene |
| Subpopulation | Indication | Comparator |
|---|---|---|
| Children aged 6 to 11 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. | Tezacaftor/Ivacaftor (zuzüglich Ivacaftor) |
Studies and Results
|
No. of studies
(best subpopulation) |
0 (Data not accepted) |
|---|---|
|
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 6 to 11 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 did not submit any direct comparative studies against the appropriate comparator therapy.
Children aged 6 to 11 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
- An additional benefit of ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor compared with the appropriate comparator therapy is not proven in the present therapeutic indication.
- Overall, additional benefit is not proven for children aged 6 to 11 years with cystic fibrosis who are heterozygous for the F508del mutation and carry a residual-function mutation on the second allele.
- Appropriate comparator therapy
- The appropriate comparator therapy was defined as follows: children aged 6 to 11 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
- Appropriate comparator therapy for ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor: tezacaftor/ivacaftor in combination with ivacaftor
Courtesy translation only, please refer to the German original.
Associated procedures
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