Following SMC collaboration with NICE on TA988: ivacaftor-tezacaftor-elexacaftor, tezacaftor-ivacaftor and lumacaftor-ivacaftor for treating cystic fibrosis
lumacaftor-ivacaftor (Orkambi®) is accepted for use within NHSScotland.
Indication under review: treatment of cystic fibrosis (CF) in patients aged 1 year and older (granules in sachet) or 6 years and older (film-coated tablets) who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
This advice applies only in the context of approved NHSScotland Patient Access Scheme (PAS) arrangements delivering the cost-effectiveness results upon which the decision was based, or PAS / list prices that are equivalent or lower.
Download detailed advice253KB (PDF)
Medicine details
- Medicine name:
- lumacaftor-ivacaftor (Orkambi)
- SMC ID:
- SMC2712
- Indication:
treatment of cystic fibrosis (CF) in patients aged 1 year and older (granules in sachet) or 6 years and older (film-coated tablets) who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
- Pharmaceutical company
- Vertex Pharmaceuticals Ltd
- BNF chapter
- Respiratory system
- Submission type
- Collaboration
- Status
- Accepted
- Date advice published
- 24 July 2024