Ivacaftor (20) – Kalydeco®
Cystic fibrosis (CF), combination treatment with ivacaftor/tezacaftor/elexacaftor in patients ≥ 12 years (heterozygous for F508del and gating mutation (incl. R117H))
Characteristics
| Start date | 01.06.2021 – Marketing authorisation: 26.04.2021 |
|---|---|
| Resolution | 19.11.2021 |
| INN | Ivacaftor |
| Brand name | Kalydeco® |
| Pharm. company | Vertex Pharmaceuticals (Germany) GmbH |
| G-BA Procedure ID | D-686 |
| 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 |
|---|---|---|
| Subjects >12 years of age and older with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a gating mutation (incl. R117H) on the second allele. | Ivacaftor |
Studies and Results
|
No. of studies
(best subpopulation) |
1 (VX18-445-104) |
|---|---|
|
Study design
(best subpopulation) |
H2H vs. ACT (off-label) |
|
Meta analysis
(best subpopulation) |
no |
- Clinical trials
- To assess the additional benefit of ivacaftor in combination with ivacaftor/tezacaftor/elexacaftor in people aged 12 years and over with cystic fibrosis who are heterozygous for the F508del mutation in the CFTR gene and carry a gatingmutation (including R117H) on the second allele, the pharmaceutical manufacturer submitted the Phase III VX18-445-104 (hereinafter ‘Study 104’) was submitted by the pharmaceutical company.
People aged 12 years and over 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 additional benefit is not proven.
- Consequently, the pharmaceutical manufacturer did not submit any study for the relevant patient population in question that would have been suitable for assessing the additional benefit of ivacaftor in combination with ivacaftor/tezacaftor/elexacaftor compared with the appropriate comparator therapy.
- Overall assessment
- Overall, additional benefit is not proven for individuals aged 12 years and over who are heterozygous for the F508del mutation in the CFTR gene and carry a gating mutation (including R117H) on the second allele.
Courtesy translation only, please refer to the German original.
Associated procedures
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