Tezacaftor / Ivacaftor (4) – Symkevi®
Cystic fibrosis (CF), combination therapy with ivacaftor in patients 6 to < 12 years (homozygous for F508del)
Characteristics
| Start date | 01.12.2020 – Marketing authorisation: 25.11.2020 |
|---|---|
| Resolution | 20.05.2021 |
| INN | Tezacaftor/Ivacaftor |
| Brand name | Symkevi® |
| Pharm. company | Vertex Pharmaceuticals (Ireland) Limited |
| G-BA Procedure ID | D-608 |
| ATC code | R07AX31 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 | 1 U 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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|
Symkevi is indicated in a combination regimen with ivacaftor tablets for the treatment of patients with cystic fibrosis (CF) aged 6 to < 12 years who are homozygous for the F508del mutation. |
| Subpopulation | Indication | Comparator |
|---|---|---|
| Children with cystic fibrosis aged 6 to < 12 years who are homozygous for the F508del mutation. | Lumacaftor/Ivacaftor |
Studies and Results
|
No. of studies
(best subpopulation) |
0 (Data not accepted) |
|---|---|
|
Study design
(best subpopulation) |
Data not accepted (Dossier: Single-arm + no comparison) |
- Clinical trials
- To demonstrate the additional benefit of TEZ/IVA + IVA in children aged 6 to < 12 years with cystic fibrosis who are homozygous for the F508del mutation, the pharmaceutical manufacturer submitted the single-arm study VX13-661-113 (hereinafter referred to as Study 113) was submitted by the pharmaceutical company to demonstrate the added benefit of TEZ/IVA + IVA in children aged 6 to < 12 years with cystic fibrosis who are homozygous for the F508del mutation.
- In addition, the pharmaceutical manufacturer submitted the results of the 8-week RCT VX16-661-115 (TEZ/IVA + IVA vs. placebo; hereinafter Study 115) and the single-arm extension study VX17-661-116 (hereinafter Study 116).
Children with cystic fibrosis aged 6 to < 12 years who are homozygous for the F508del mutation
- For children with cystic fibrosis aged 6 to < 12 years who are homozygous for the F508del mutation, the additional benefit of tezacaftor/ivacaftor in combination with ivacaftor compared with the appropriate comparator therapy is not proven.
- Overall, therefore, additional benefit is not proven for patients aged 6 to < 12 years with cystic fibrosis who are homozygous for the F508del mutation in the CFTR gene.
- Overall assessment
- Overall, additional benefit is not proven for patients aged 6 years or older but under 12 years with cystic fibrosis who are homozygous for the F508del mutation in the CFTR gene.
Courtesy translation only, please refer to the German original.
Associated procedures
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