Eliglustat (Cerdelga) is a selective glucosylceramide synthase inhibitor indicated for the long-term treatment of Type 1 Gaucher disease (GD1) in adults. Its efficacy and effectiveness are highly dependent on an individual's metabolic status and comorbidities. Therefore, it is essential to strictly follow the dosing regimen based on CYP2D6 metabolic phenotype and adjust the dose for special populations.

How to Use Eliglustat (Cerdelga)

Dosage and Administration Frequency

Extensive Metabolizers (EMs): 84 mg orally, twice daily.

Intermediate Metabolizers (IMs): 84 mg orally, twice daily.

Poor Metabolizers (PMs): 84 mg orally, once daily.

Administration Precautions

Swallow Whole: Capsules must not be crushed, dissolved, or opened.

Dietary Effects: The medication can be taken with or without food, but grapefruit and its products (strong CYP3A inhibitors) should be avoided.

Management of Missed Doses: If a dose is missed, take the next dose at the originally scheduled time; do not make up for the missed dose.

Dosage Adjustment of Eliglustat (Cerdelga)

Adjustment During Concomitant Medication Use

If co-administered with strong/moderate CYP2D6 or CYP3A inhibitors (e.g., fluconazole, paroxetine), the dose must be reduced to 84 mg once daily.

Strong CYP3A inhibitors (e.g., ketoconazole) are contraindicated. No dose adjustment is needed for CYP2D6 inhibitors (since PMs lack CYP2D6 activity).

Hepatic Impairment

Contraindicated in patients with moderate to severe hepatic impairment; also contraindicated in patients with mild hepatic impairment who are co-administered CYP2D6 inhibitors.

Contraindicated in patients with any degree of hepatic impairment.

Renal Impairment

Use should be avoided in patients with end-stage renal disease (ESRD); no dose adjustment is required for mild to moderate renal impairment.

Use should be avoided in patients with any degree of renal impairment.

Eliglustat (Cerdelga) Use in Special Populations

Pregnancy and Lactation

Pregnant Women: Uncontrolled Gaucher disease may increase the risk of miscarriage. The therapeutic benefits and potential fetal risks must be carefully weighed. Animal studies have shown that high doses of eliglustat can cause fetal skeletal malformations.

Lactation: The concentration of eliglustat in rat milk is higher than that in plasma. It is recommended to weigh the necessity of breastfeeding against discontinuing the medication.

Pediatric and Geriatric Populations

Pediatric Patients: The effectiveness of eliglustat has not been established, so its use is not recommended.

Geriatric Patients: Clinical data are limited, but no clear age-related differences have been observed.

Patients with Cardiac Diseases

Use should be avoided in patients with pre-existing QT interval prolongation, heart failure, bradycardia, or those taking Class IA/III antiarrhythmic drugs, as the medication may increase the risk of arrhythmias.