Miglustat (Zavesca) is a glucosylceramide synthase inhibitor that was first approved in the United States in 2003. As a monotherapy, this medication is indicated for adult patients with mild to moderate Type 1 Gaucher disease who are unable to receive enzyme replacement therapy (e.g., due to allergy, hypersensitivity, or poor venous access).

How to Use Miglustat (Zavesca)

Recommended Dosage Standards

Adult Dosage: 100 mg orally three times daily, with regular intervals between administrations.

Dosage Adjustment: Some patients may need to have their dosage reduced to 100 mg once or twice daily due to adverse reactions such as tremors or diarrhea.

Administration Time Requirements

Oral administration should be at approximately the same times each day, and the medication can be taken with or without food.

Capsules must be swallowed whole; they should not be opened, crushed, or chewed.

If vomiting occurs after taking the medication, no additional dose needs to be administered.

If a dose is missed, the next capsule should be taken at the originally scheduled next time; do not take an extra dose to make up for the missed one.

Dosage Adjustment Scenarios for Miglustat (Zavesca)

Dosage Adjustment for Adverse Reactions

Tremors: Approximately 30% of patients report developing tremors or worsening of existing tremors, which usually occur within the first month of treatment. The dosage should be reduced to alleviate tremors; if tremors do not improve within a few days after dosage reduction, treatment should be discontinued.

Diarrhea and Weight Loss: In clinical trials, approximately 85% of patients experienced diarrhea and 65% experienced weight loss. These symptoms can be managed through dietary adjustments (e.g., reducing intake of carbohydrates such as sucrose and lactose), taking the medication between meals, or using antidiarrheal agents (e.g., loperamide).

Dosage Adjustment for Patients with Renal Impairment

Mild Renal Impairment (creatinine clearance 50–70 mL/min/1.73 m²): The initial dosage is 100 mg twice daily.

Moderate Renal Impairment (creatinine clearance 30–50 mL/min/1.73 m²): The initial dosage is 100 mg once daily.

Severe Renal Impairment (creatinine clearance < 30 mL/min/1.73 m²): Use is not recommended.

Miglustat (Zavesca) Use in Special Populations

Pregnant and Lactating Women

Pregnancy: Based on animal study results, administration of miglustat to pregnant women may cause embryo-fetal harm.

Lactation: Miglustat is likely to be present in human milk. Due to the potential risk of serious adverse reactions in breastfed infants, women are advised not to breastfeed during miglustat treatment.

Elderly Patients

Clinical studies of miglustat did not include a sufficient number of patients aged 65 years and older to determine whether their responses differ from those of younger patients.

Elderly patients are more likely to have decreased renal function, so dosage selection should be cautious, usually starting from the lower end of the dosage range.

Patients of Reproductive Age

Based on animal studies, miglustat may affect fertility.

Studies in male patients have shown that miglustat can cause a decrease in fertility, which is reversible.