Glucosylceramide synthase inhibitor (substrate reduction therapy)
Eliglustat
Brand names: Cerdelga
Adult dose
Dose: 84mg BD (extensive/intermediate metabolisers); 84mg OD (poor metabolisers) — based on CYP2D6 phenotype
Route: Oral
Frequency: OD–BD
Clinical pearls
- Type 1 Gaucher disease — alternative to enzyme replacement (imiglucerase, velaglucerase) per UK Lysosomal Storage Disorder Service
- NICE TA recommendations; specialist metabolic centre
- CYP2D6 genotyping mandatory before prescribing
Contraindications
- CYP2D6 ultra-rapid metabolisers (insufficient drug exposure)
- Concurrent strong CYP2D6 + CYP3A4 inhibitors
- Severe hepatic impairment
- Hypersensitivity
Side effects
- Headache
- Dizziness
- Fatigue
- Nausea
- Diarrhoea
- Arthralgia
- QT prolongation
- Hepatotoxicity
Interactions
- CYP2D6 inhibitors (paroxetine, fluoxetine — avoid)
- CYP3A4 inhibitors
- QT-prolonging drugs
Monitoring
- Disease parameters (haematology, splenomegaly, bone)
- ECG (QT)
- LFTs
- CYP2D6 genotype documented
Reference: BNF; NICE TA354; UK LSD National Service; BIMDG; SmPC; https://bnf.nice.org.uk/drugs/eliglustat/. Verify against your local formulary and the latest BNF before prescribing.
Related
Curated clinical cross-links plus same-class fallbacks.
Calculators
- DAPT Score for Dual Antiplatelet Therapy Duration · Antiplatelet Therapy
- ACC/AHA Pooled Cohort Equations (ASCVD Risk) · Cardiovascular Risk
- SMART Risk Score for Recurrent CVD · Cardiovascular Risk
- IABP Timing Assessment · Mechanical Circulatory Support
- DAPT Decision Tool (Ticagrelor vs Clopidogrel) · Antiplatelet Therapy
- PCSK9 Inhibitor Eligibility Assessment · Lipid Management
Pathways
- Sepsis Screening and Sepsis Six · UK Sepsis Trust; NICE NG51; Surviving Sepsis Campaign 2021
- Unintentional Weight Loss Workup · NICE NG12; BSG
- Chronic Fatigue Workup · NICE NG206; BMJ Best Practice
- Lymphadenopathy Workup · NICE NG12; BMJ Best Practice
- Pre-op Medical Clearance · NICE NG45; ESC 2022
- Secondary Hypertension Workup · NICE NG136; ESH 2023