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 |
|---|---|
| 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) |
| DDD | 1 U O |
| Therapeutic area | Metabolic diseases Orphan (turnover limit) |
| Reason for procedure | New therapeutic indication |
| Specialty | Bundling |
Studies and Results
- 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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