Tezacaftor / Ivacaftor (5) – Symkevi®
Cystic fibrosis (CF), combination therapy with ivacaftor in patients 6 to < 12 years (heterozygous for F508del and RF mutation)
Characteristics
| Start date | 01.12.2020 – Marketing authorisation: 25.11.2020 |
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| Resolution | 20.05.2021 |
| INN | Tezacaftor/Ivacaftor |
| Brand name | Symkevi® |
| Pharm. company | Vertex Pharmaceuticals (Ireland) Limited |
| G-BA Procedure ID | D-609 |
| 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 heterozygous for the F508del mutation and have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T. |
| Subpopulation | Indication | Comparator |
|---|---|---|
| Children with cystic fibrosis aged 6 to < 12 years who are heterozygous for the F508del mutation and have one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T. | Best-Supportive-Care |
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
- In addition, the pharmaceutical manufacturer provided the results of the 8-week RCT VX16-661-115 (TEZ/IVA + IVA vs. IVA; hereinafter referred to as Study 115) and the single-arm extension study VX17-661-116 (hereinafter referred to as Study 116).
Children with cystic fibrosis aged 6 to < 12 years who are heterozygous for the F508del mutation and carry one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272–26A→G and 3849+10kbC→T
- For children with cystic fibrosis aged 6 to < 12 years who are heterozygous for the F508del mutation and carry one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272–26A→G, and 3849+10kbC→T, the additional benefit of tezacaftor/ivacaftor in combination with ivacaftor compared with the appropriate comparator therapy is not proven.
- In its dossier for the assessment of the additional benefit of TEZ/IVA + IVA, the pharmaceutical manufacturer does not present any direct comparative studies against the appropriate comparator therapy, Best Supportive Care (BSC).
- Furthermore, no indirect comparisons were submitted to address the issues raised in the benefit assessment.
- The single-arm study 113 is not relevant to the present benefit assessment, as no data are available for an assessment of TEZ/IVA + IVA compared with the appropriate comparator therapy.
- Consequently, the pharmaceutical manufacturer did not submit any study for this patient population that would have been suitable for assessing the additional benefit of TEZ/IVA + IVA compared with the appropriate comparator therapy.
- Overall assessment
- For patients aged 6 years or older but under 12 years with cystic fibrosis who are heterozygous for the F508del mutation and carry one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272–26A→G, and 3849+10kbC→T, additional benefit is not proven.
Courtesy translation only, please refer to the German original.
Associated procedures
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