Ivacaftor (27) – Kalydeco®
Cystic fibrosis (CF), F508del mutation, heterozygous and other or unknown mutation, ≥ 6 to ≤ 11 years, combination with ivacaftor / tezacaftor / elexacaftor
Characteristics
| Start date | 15.02.2022 – Marketing authorisation: 07.01.2022 |
|---|---|
| Resolution | 04.08.2022 |
| INN | Ivacaftor |
| Brand name | Kalydeco® |
| Pharm. company | Vertex Pharmaceuticals (Germany) GmbH |
| G-BA Procedure ID | D-797 |
| ATC code | R07AX02 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 | 0.3 g O |
| 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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|
Kalydeco tablets are used as part of a combination treatment with ivacaftor/tezacaftor/elexacaftor tablets for the treatment of adults, adolescents and children aged and children aged 6 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the 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 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 (BSC) |
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 in combination with ivacaftor/tezacaftor/elexacaftor for the treatment of children aged 6 to 11 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) and no residual function mutation, or in whom the mutation on the second allele is unknown (other mutations), the pharmaceutical manufacturer has not submitted any study data.
Children aged 6 to 11 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)
- An additional benefit is not proven.
- Overall, the pharmaceutical manufacturer did not submit any study for the present therapeutic indication that would have been suitable for assessing the additional benefit of ivacaftor in combination with ivacaftor/tezacaftor/elexacaftor compared with the appropriate comparator therapy.
- An additional benefit of ivacaftor in combination with ivacaftor/tezacaftor/elexacaftor compared with the appropriate comparator therapy is not proven.
- Overall assessment
- Taking into account the available evidence on the medical benefit of ivacaftor in combination with ivacaftor/tezacaftor/Elexacaftor, the progressive course of the disease and the statements from medical societies regarding the current reality of care, Ivacaftor in combination with Ivacaftor/Tezacaftor/Elexacaftor may represent a relevant treatment option for individual patients aged 6 to 11 years who are heterozygous for the F508del mutation in the CFTR gene and carry an unknown or other mutation on the second allele.
Courtesy translation only, please refer to the German original.
Associated procedures
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