Advice

following a full submission considered under the orphan medicine process

lumacaftor, ivacaftor (Orkambi®) is not recommended for use within NHS Scotland.

Indication under review: treatment of cystic fibrosis (CF) in patients aged 12 years and older who are homozygous for the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene.

Lumacaftor-ivacaftor, compared to placebo, significantly increased percent predicted forced expiratory volume in one second (ppFEV1) by less than 3% at six months and reduced the annual rate of pulmonary exacerbations in patients with CF homozygous for the F508del mutation of 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 and Engagement (PACE) meeting.

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Medicine details

Medicine name:
lumacaftor-ivacaftor (Orkambi)
SMC ID:
1136/16
Indication:
Treatment of cystic fibrosis (CF) in patients aged 12 years and older who are homozygous for the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene.
Pharmaceutical company
Vertex Pharmaceuticals Ltd
BNF chapter
Respiratory system
Submission type
Full submission
Status
Not recommended
Date advice published:
09 May 2016