following a full submission assessed under the ultra orphan medicine process
ivacaftor (Kalydeco®) is not recommended for use within NHS Scotland.
Indication under review: treatment of children with cystic fibrosis (CF) aged 2 years and older and weighing less than 25kg who have one of the following gating (class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
In an open-label single-arm study, acceptable safety was demonstrated in children aged 2 to 5 years.
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 and Engagement (PACE) meeting.
Download detailed advice212KB (PDF)
Medicine details
- Medicine name:
- ivacaftor (Kalydeco)
- SMC ID:
- 1134/16
- Indication:
- Treatment of children with cystic fibrosis (CF) aged 2 years and older and weighing less than 25kg who have one of the following gating (class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
- Pharmaceutical company
- Vertex Pharmaceuticals Ltd
- BNF chapter
- Respiratory system
- Submission type
- Full
- Status
- Not recommended
- Date advice published
- 09 May 2016