Advice

following a full submission considered under the orphan process:

tezacaftor-ivacaftor (Symkevi®) is not recommended for use within NHSScotland.

Indication under review: In a combination regimen with ivacaftor 150mg tablets for the treatment of patients with cystic fibrosis (CF) aged 12 years and older who are homozygous for the F508del mutation or 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.

In phase III studies in patients ≥12 years of age with cystic fibrosis who were homozygous for the F508del CFTR mutation or heterozygous for the F508del CFTR mutation and a second allele with a CFTR mutation with residual function, tezacaftor-ivacaftor was superior to placebo for absolute change in the percent predicted forced expiratory volume in one second (ppFEV1) from the baseline.

The submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present a sufficiently robust clinical and economic analysis to gain acceptance by SMC.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.

Medicine details

Medicine name:
tezacaftor-ivacaftor (Symkevi)
SMC ID:
SMC2183
Indication:

In a combination regimen with ivacaftor 150 mg tablets for the treatment of patients with cystic fibrosis (CF) aged 12 years and older who are homozygous for the F508del mutation or 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.

Pharmaceutical company
Vertex Pharmaceuticals Ltd
BNF chapter
Respiratory system
Submission type
Full
Status
Not recommended
Date advice published:
12 August 2019