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

Studies and Results

  • 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

Tezacaftor / Ivacaftor (5) Symkevi® Vertex Pharmaceuticals (Ireland) Limited Metabolic diseases Cystic fibrosis (CF), combination therapy with ivacaftor in patients 6 to < 12 years (heterozygous for F508del and RF mutation) 50 100% additional benefit not proven Orphan (turnover limit)
Tezacaftor / Ivacaftor (4) Symkevi® Vertex Pharmaceuticals (Ireland) Limited Metabolic diseases Cystic fibrosis (CF), combination therapy with ivacaftor in patients 6 to < 12 years (homozygous for F508del) 470 100% additional benefit not proven Orphan (turnover limit)
Tezacaftor / Ivacaftor (2) Symkevi® Vertex Pharmaceuticals Metabolic diseases Cystic fibrosis (CF), combination treatment with ivacaftor in patients ≥ 12 years (homozygous for F508del) 2,400 100% additional benefit not proven Orphan (turnover limit)
Tezacaftor / Ivacaftor (3) Symkevi® Vertex Pharmaceuticals Metabolic diseases Cystic fibrosis (CF), combination treatment with ivacaftor in patients ≥ 12 years (heterozygous for F508del) 200–300 100% additional benefit not proven Orphan (turnover limit)
Tezacaftor / Ivacaftor (1) Symkevi® Vertex Pharmaceuticals (Germany) GmbH Metabolic diseases Cystic fibrosis (CF), F508del mutation, ≥ 12 years 0
2,600–2,700 		91% considerable additional benefit Orphan repealed


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