Eliglustat
CERDELGA
What it isGlucosylceramide synthase inhibitor; Gaucher disease type 1.
Why this oneOral substrate reduction rather than biweekly enzyme infusion.
What limits it⚑ CYP2D6 genotype determines dose, and poor metabolizers cannot use it at all. QT prolongation; many contraindicated interactions.
FDA-approved for
- Gaucher disease type 1 (2014)
FDA label
Read the full label on DailyMed ↗
| FDA dosing | Population | Start | Target | Max |
|---|---|---|---|---|
| Gaucher disease type 1 | CYP2D6 extensive or intermediate metabolizer | 84 mg BID | 84 mg BID | 84 mg BID |
| Gaucher disease type 1 | CYP2D6 poor metabolizer | 84 mg QD | 84 mg QD | 84 mg QD |
Renal EMs: avoid in end-stage renal disease; otherwise use per CYP2D6 metabolizer status
Hepatic Contraindicated in IMs/PMs with any hepatic impairment and in EMs with moderate-severe impairment (or mild impairment plus a CYP2D6 inhibitor); genotype-dependent
Dose frequency set by CYP2D6 metabolizer status and interacting CYP2D6/CYP3A inhibitors
Instructions
- Select patients with an FDA-cleared CYP2D6 genotype test.
- Swallow capsules whole; do not crush, dissolve, or open.
- Avoid grapefruit or grapefruit juice.
- Reduce to 84 mg once daily in EMs/IMs taking CYP2D6 or CYP3A inhibitors.
Contraindications
- EMs taking a strong/moderate CYP2D6 inhibitor with a strong/moderate CYP3A inhibitor
- EMs with moderate or severe hepatic impairment, or mild hepatic impairment plus a strong/moderate CYP2D6 inhibitor
- IMs taking a strong/moderate CYP2D6 inhibitor with a strong/moderate CYP3A inhibitor, or a strong CYP3A inhibitor
- IMs or PMs with any degree of hepatic impairment
- PMs taking a strong CYP3A inhibitor
Cautions
- ECG interval prolongation (PR, QTc, QRS) and potential for cardiac arrhythmias at elevated plasma concentrations
- Avoid in pre-existing cardiac disease or long QT syndrome combined with Class IA/III antiarrhythmics
Adverse reactions
- Fatigue
- Headache
- Nausea
- Diarrhea
- Back pain
- Pain in extremities
- Upper abdominal pain
Pregnancy Insufficient human data; developmental abnormalities in rats at ~6x human dose.
Lactation No human data; eliglustat present in rat milk.
NO BOXED WARNINGS
Principal riskCYP2D6 genotype determines dose; poor metabolizers cannot use it
Cost, est. cash$1,000–$50,000+/month
Generic entry2021
Classes and tags
Clinical profile
Legacy pregnancy categoryUnknown / historical label unavailable
Defining liabilityGenerally tolerated at therapeutic doses; clinically important risks are agent-specific and increase with interactions or organ impairment.
Mechanism of action: Eliglustat ballicule
