CFTR corrector + potentiator
Lumacaftor with ivacaftor
Brand names: Orkambi
Adult dose
Dose: Per BNF/SmPC; weight/age-based with food
Route: PO
Frequency: BD
Clinical pearls
- Cystic fibrosis homozygous F508del; largely superseded by ivacaftor-tezacaftor-elexacaftor (Kaftrio) where eligible
Contraindications
- Severe hepatic impairment without adjustment
Side effects
- Dyspnoea (lumacaftor-related, esp. low FEV1)
- Diarrhoea
- Hepatotoxicity
- Hypertension
- Cataract (paediatric)
Interactions
- Strong CYP3A4 inducer (lumacaftor) — many
- Reduces hormonal contraceptive efficacy
- Statins / warfarin / carbamazepine / midazolam
Monitoring
- LFTs
- BP
- Ophthalmic (children)
Reference: BNF; NICE TA787; SmPC; https://bnf.nice.org.uk/drugs/lumacaftor-with-ivacaftor/. Verify against your local formulary and the latest BNF before prescribing.
Related
Curated clinical cross-links plus same-class fallbacks.
Pathways
- Acute Asthma in Adults · BTS/SIGN British Guideline on Asthma 2019; NICE NG80
- Pulmonary Embolism Assessment · NICE NG158; ESC 2019 PE Guidelines
- Acute Exacerbation of COPD (AECOPD) · NICE NG115; GOLD 2024
- Spontaneous Pneumothorax (Adult) · BTS Pleural Disease 2023
- Atypical Pneumonia (Legionella / Mycoplasma / Chlamydophila) · BTS 2023; IDSA
- COPD Exacerbation Management · NICE NG115 / GOLD 2024