Eliglustat (Cerdelga) is a glucosylceramide synthase inhibitor used for the long-term treatment of Type 1 Gaucher Disease in adults, with eliglustat as its active ingredient.

What Are the Side Effects of Eliglustat (Cerdelga)?

Common Adverse Reactions

Adverse reactions with an incidence of ≥10% include fatigue, headache, nausea, diarrhea, back pain, limb pain, and upper abdominal pain. Most of these symptoms are mild to moderate and usually do not require treatment interruption.

Serious Side Effects of Eliglustat (Cerdelga) That Require Vigilance

1. Long QT Syndrome

(1) Concurrent use of Class IA (e.g., quinidine, procainamide) or Class III (e.g., amiodarone, sotalol) antiarrhythmic drugs.

(2) If patients experience new symptoms such as palpitations, syncope, or dizziness, they should inform their doctor immediately.

2. Serious Risks Caused by Drug Interactions

(1) Concurrent use with CYP2D6 or CYP3A inhibitors may increase the blood concentration of eliglustat, raising the risk of cardiotoxicity.

(2) Concurrent use with strong CYP3A inducers may decrease the concentration of eliglustat, reducing its therapeutic efficacy.

3. Patients with Hepatic Impairment

(1) Extensive Metabolizer (EM) patients with moderate to severe hepatic impairment (Child-Pugh Class B or C) are contraindicated.

(2) EM patients with mild hepatic impairment (Child-Pugh Class A) who are also using strong or moderate CYP2D6 inhibitors are contraindicated.

(3) Intermediate Metabolizer (IM) and Poor Metabolizer (PM) patients with hepatic impairment of any severity are contraindicated.

Precautions for Eliglustat (Cerdelga) Administration

1. Patient Selection and Genetic Testing

(1) Before using eliglustat, patients must undergo FDA-approved testing to determine their CYP2D6 metabolic phenotype.

(2) It is recommended for patients with CYP2D6 Extensive Metabolizer (EM), Intermediate Metabolizer (IM), or Poor Metabolizer (PM) phenotypes.

(3) It is not recommended for patients with CYP2D6 Ultra-Rapid Metabolizer (URM) phenotype, as they may fail to achieve therapeutic concentrations.

(4) There is no specific dosage recommendation for patients with undetermined CYP2D6 genotype (unknown metabolic phenotype).

2. Administration Guidelines

(1) Swallow the capsules whole; do not crush, dissolve, or open the capsules.

(2) It can be taken with food or on an empty stomach.

(3) Avoid consuming grapefruit or drinking grapefruit juice (a strong CYP3A inhibitor).

(4) If a dose is missed, take the prescribed dose at the next regular time; do not double the dose.

3. Information to Inform the Doctor

(1) All medications currently in use, including prescription drugs, over-the-counter drugs, and herbal supplements.

(2) History of heart disease, including heart failure, recent heart attack, bradycardia, heart block, or arrhythmia.

(3) Personal or family history of long QT syndrome.

(4) Hepatic and renal function status.

(5) Plans for pregnancy or breastfeeding.