Eliglustat (Cerdelga) is a glucosylceramide synthase inhibitor that received its first approval in the United States in 2014. As a long-acting therapeutic agent for Type 1 Gaucher disease, it provides an important oral treatment option for patients with specific metabolic phenotypes.

How to Use Eliglustat (Cerdelga)

Patient Selection Criteria

When selecting patients with Type 1 Gaucher disease, the choice must be based on the patient’s CYP2D6 metabolic status.

It is recommended to use FDA-approved testing to determine the CYP2D6 genotype.

Recommended Dosages Based on Metabolic Status

Extensive Metabolizers (EMs) and Intermediate Metabolizers (IMs): 84 mg taken orally twice daily.

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

Important Administration Instructions

Swallow the capsules whole, preferably with water. Do not crush, dissolve, or open the capsules.

Eliglustat can be taken with or without food.

Avoid consuming grapefruit or drinking grapefruit juice.

If a dose of eliglustat is missed, take the prescribed dose at the next scheduled time. Do not double the next dose.

For patients currently receiving imiglucerase, velaglucerase alfa, or taliglucerase alfa treatment, eliglustat can be initiated 24 hours after the last enzyme replacement therapy (ERT) administration.

Dose Adjustment for Eliglustat (Cerdelga)

Management of Special Situations

Cardiac Event Management: When plasma concentrations of eliglustat are significantly elevated, it may cause prolongation of electrocardiogram (ECG) intervals (PR, QTc, and QRS), increasing the risk of arrhythmias.

Avoid use in patients with pre-existing heart conditions, including congestive heart failure, recent acute myocardial infarction, bradycardia, heart block, and ventricular arrhythmias.

Long QT Syndrome

Concomitant use with Class IA (e.g., quinidine, procainamide) and Class III (e.g., amiodarone, sotalol) antiarrhythmic drugs is not recommended.

Infection Management: If a severe infection occurs, timely evaluation and appropriate measures should be taken.

Use of Eliglustat (Cerdelga) in Special Populations

Patients with Renal Impairment

Extensive Metabolizers (EMs): Avoid use in patients with end-stage renal disease (ESRD) (estimated creatinine clearance [eCLcr] less than 15 mL/min, with or without dialysis).

No dose adjustment is required for patients with mild, moderate, or severe renal impairment.

Intermediate Metabolizers (IMs) and Poor Metabolizers (PMs): Avoid use of eliglustat in patients with any degree of renal impairment.

Patients with Hepatic Impairment

Extensive Metabolizers (EMs): Contraindicated in patients with severe (Child-Pugh Class C) hepatic impairment.

Contraindicated in patients with moderate (Child-Pugh Class B) hepatic impairment.

Contraindicated in patients with mild (Child-Pugh Class A) hepatic impairment who are taking strong or moderate CYP2D6 inhibitors.

Intermediate Metabolizers (IMs) and Poor Metabolizers (PMs): Contraindicated in patients with any degree of hepatic impairment.

Lactation

There are no human data available regarding whether eliglustat is present in human milk, its effects on breastfed infants, or its effects on milk production.

When a drug is present in animal milk, it is likely to be present in human milk.

Consider the developmental and health benefits of breastfeeding, the mother’s clinical need for eliglustat, and any potential adverse effects of eliglustat or the underlying maternal condition on the breastfed child.