lumacaftor-ivacaftor (Orkambi)

following a full submission considered under the orphan equivalent process:

lumacaftor-ivacaftor (Orkambi®) is not recommended for use within NHSScotland.

Indication under review: the treatment of cystic fibrosis in patients aged 6 years and older (tablets) and aged 2 to 5 years (granules) who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

lumacaftor-ivacaftor, compared with placebo, improved measures of lung function in patients with cystic fibrosis who were homozygous for the F508del mutation in the CFTR gene.

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