Ivacaftor (22) – Kalydeco®
Cystic fibrosis (CF), combination treatment with ivacaftor/tezacaftor/elexacaftor in patients ≥ 12 years (heterozygous for F508del and others or unknown mutation)
Characteristics
| Start date | 01.06.2021 – Marketing authorisation: 26.04.2021 |
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| Resolution | 19.11.2021 |
| INN | Ivacaftor |
| Brand name | Kalydeco® |
| Pharm. company | Vertex Pharmaceuticals (Germany) GmbH |
| G-BA Procedure ID | D-690 |
| 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 | 0.15 mg 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 indicated: in a combination regimen with ivacaftor /tezacaftor /elexacaftor tablets for the treatment of adults and adolescents aged 12 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene |
| Subpopulation | Indication | Comparator |
|---|---|---|
| E) | Subjects 12 years of age and older with cystic fibrosis (CF) who are heterozygous for the F508del mutation are heterozygous for the F508del mutation in the CFTR gene and have a mutation in the second allele that is not a minimal function, gating (including R117H) or residual function mutation, or in which the mutation in the second allele is unknown (other mutations). | Best-Supportive-Care (BSC) |
Studies and Results
|
No. of studies
(best subpopulation) |
0 (Data not accepted) |
|---|---|
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Study design
(best subpopulation) |
Data not accepted (Dossier: H2H vs. non-ACT + no ITC) |
- Clinical trials
- To assess the additional benefit of IVA + IVA/TEZ/ELX for the treatment of people aged 12 years and over with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and who have 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 did not submit any study data.
Individuals aged 12 years and over with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and who have 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 IVA + IVA/TEZ/ELX compared with the appropriate comparator therapy.
- An additional benefit of IVA + IVA/TEZ/ELX compared with the appropriate comparator therapy is therefore not proven.
- Overall assessment
- Taking into account the available evidence on the medical benefit of IVA + IVA/TEZ/ELX, 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 represent a relevant treatment option for patients aged 12 years and over who are heterozygous for the F508del mutation in the CFTR gene and have an unknown or other mutation on the second allele.
Courtesy translation only, please refer to the German original.
Associated procedures
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