Ivacaftor (26) – Kalydeco®
Cystic fibrosis (CF), F508del mutation, heterozygous and RF 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-796 |
| 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 residual function mutation on the second allele. | Tezacaftor/Ivacaftor (plus Ivacaftor) |
Studies and Results
|
No. of studies
(best subpopulation) |
0 (no data submitted) |
|---|---|
|
Study design
(best subpopulation) |
no data submitted (Dossier: Evidence transfer) |
- Clinical trials
- To assess the additional benefit of ivacaftor in combination with ivacaftor/tezacaftor/elexacaftor 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.
- 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 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 residual-function mutation on the second allele
- An additional benefit is not proven.
- Overall, additional benefit is not proven for children aged 6 to 11 with cystic fibrosis who are heterozygous for the F508del mutation and carry a residual-function mutation on the second allele.
- Overall assessment
- On balance, additional benefit is not proven for children aged 6 to 11 with cystic fibrosis who are heterozygous for the F508del mutation and carry a residual-function mutation on the second allele.
Courtesy translation only, please refer to the German original.
Associated procedures
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