Ivacaftor / Tezacaftor / Elexacaftor (8) – Kaftrio®
Cystic fibrosis, F508del mutation, heterozygous and gating 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-775 |
| 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 |
|---|---|---|
| C) | Children aged 6 to 11 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a gating mutation (including R117H) on the second allele. | 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
- The key study, VX18-445-104, which the pharmaceutical manufacturer cites in its argument for extending the additional benefit to patients aged 12 and over, was already deemed unsuitable in the context of the benefit assessments for this age group due to the study duration being insufficient.
Children aged 6 to 11 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a gating mutation (including R117H) on the second allele
- For the assessment of 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 gating mutation (including R117H) on the second allele, the pharmaceutical manufacturer has not submitted any direct comparative studies against the appropriate comparator therapy.
- It is not possible to extrapolate the additional benefit to the population in the present therapeutic indication, based on the information provided by the pharmaceutical manufacturer, either to patients with a different mutation type or to older patients.
- Overall, for children aged 6 to 11 years with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a gating mutation (including R117H) on the second allele, additional benefit is therefore not proven.
Courtesy translation only, please refer to the German original.
Associated procedures
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