Advice
Following a resubmission:
ivacaftor (Kalydeco®) is not recommended for use within NHS Scotland.
Indication under review: treatment of cystic fibrosis (CF) in patients age 6 years and older who have a G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Ivacaftor has demonstrated superiority over placebo measured by absolute change in forced expiratory volume in one second (FEV1) % predicted in two phase III, double-blind randomised studies.
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 economic assessment to gain acceptance by SMC.
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Medicine details
- Medicine name:
- ivacaftor (Kalydeco)
- SMC ID:
- 827/12
- Indication:
- For the treatment of cystic fibrosis (CF) in patients age 6 years and older who have a G551D mutation in the CFTR (cystic fibrosis transmembrane conductance regulator) gene.
- Pharmaceutical company
- Vertex Pharmaceuticals Ltd
- BNF chapter
- Respiratory system
- Submission type
- Resubmission
- Status
- Not recommended
- Date advice published
- 10 June 2013