lumacaftor-ivacaftor (Orkambi) -

lumacaftor-ivacaftor (Orkambi)
Information type: Guidance
Source: Scottish Medicines Consortium
Specialities: Paediatric and neonatal medicine | Respiratory disorders
Summary
The SMC does not recommend lumacaftor-ivacaftor for use within NHS Scotland, for the 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.
UKMi comment
The submitting company did not did not present a sufficiently robust clinical and economic analysis to gain acceptance by the SMC.
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