Eliglustat (Cerdelga) is a glucosylceramide synthase inhibitor. It has been approved by the U.S. FDA for the treatment of adult patients with Type 1 Gaucher disease. Its eligible recipients must be CYP2D6 extensive metabolizers, intermediate metabolizers, or poor metabolizers, and this status must be verified through FDA-certified testing.

What Are the Side Effects of Eliglustat (Cerdelga)?

Patient Response Characteristics in the Initial Treatment Phase

Joint pain: Incidence rate of 45%.

Headache: Incidence rate of 40%.

Sore throat: Incidence rate of 10%.

Nausea: Incidence rate of 10%.

Flatulence: Incidence rate of 10%.

Responses of Patients Switched to Eliglustat After Previous Enzyme Replacement Therapy

Fatigue: Incidence rate of 14%.

Headache: Incidence rate of 13%.

Nausea: Incidence rate of 12%.

Diarrhea: Incidence rate of 12%.

Back pain: Incidence rate of 12%.

Severe Side Effects of Eliglustat (Cerdelga)

Risk of ECG Changes and Arrhythmias

When the plasma concentration of eliglustat increases significantly, it may cause prolongation of PR, QTc, and QRS intervals, significantly increasing the risk of arrhythmias.

The following patient groups require special attention: those with pre-existing heart disease (such as heart failure, recent acute myocardial infarction).

Bradycardia or heart block.

Ventricular arrhythmias.

Patients with long QT syndrome.

Special Risks for Patients with Hepatic Impairment

Extensive metabolizers: Moderate to severe hepatic impairment is an absolute contraindication.

Intermediate metabolizers and poor metabolizers: Hepatic impairment of any degree is a contraindication.

Precautions for Eliglustat (Cerdelga)

Patient Screening and Individualized Treatment

Before treatment, the CYP2D6 genotype must be determined through FDA-approved testing.

Ultra-rapid metabolizers may not reach effective therapeutic concentrations.

For patients whose genotype cannot be determined, no specific dosage can be recommended.

Special Considerations for Patients with Renal Impairment

Extensive metabolizers: Use should be avoided in patients with end-stage renal disease (eCLcr < 15 mL/min).

No dosage adjustment is required for mild, moderate, or severe renal impairment.

Intermediate metabolizers and poor metabolizers: Use should be avoided in patients with renal impairment of any degree.

Medication Adherence Education

Swallow the capsules whole; do not crush, dissolve, or open them.

The medication can be taken with food or on an empty stomach.

Avoid consuming grapefruit or drinking grapefruit juice.

If a dose is missed, do not double the dose at the next scheduled time.