Adult Dosing
Mild to moderate type 1 Gaucher disease
- 100 mg PO tid at regular intervals
- In case of adverse effects such as diarrhea or tremor, may decrease to 100 mg PO qd-bid
Pediatric Dosing
- Safety and effectiveness in pediatric patients have not been established
[Outline]
Renal Dose Adjustment (Based on CrCl)
- Mild renal impairment (50-70 mL/min): 100 mg PO bid
- Moderate renal impairment (30-50 mL/min): 100 mg PO qd
- Severe renal impairment (<30 mL/min): Contraindicated
Hepatic Dose Adjustment
- Hepatic impairment: No dose adjustments
See Supplemental Patient Information
- Treatment should be directed by physicians with experience in the management of Gaucher disease
- Therapy may cause peripheral neuropathy. Patients receiving miglustat must undergo baseline and repeat neurological evaluations at about 6-month intervals
- Discontinuation of therapy is to be considered if symptoms such as numbness and tingling occur
- Therapy may cause tremor or exacerbation of existing tremor; mostly occurs within the first month of therapy and resolves between 1-3 months during treatment. Dose reduction or discontinuation of therapy may be needed to ameliorate the tremor
- Incidence of diarrhea and weight loss is common during treatment. Weight loss usually occurs during the first 12 months of treatment. Diarrhea was noted to decrease over time with continued treatment. Patients presenting with diarrhea may be advised to avoid high carbohydrate content foods during treatment
- Patients with persistent GI events that continue during miglustat therapy should be evaluated for any significant underlying GI disease
- There have been reports of decreased platelet counts during treatment without association with bleeding
- Male patients, before seeking to conceive, should cease miglustat therapy and maintain reliable contraceptive method for 3 months thereafter
Cautions: Use cautiously in
- GI disorder
- Elderly patients
Supplemental Patient Information
- Advise patients to avoid high carbohydrate content food during the treatment
- Instruct patients to promptly report any numbness, pain, or tingling in the hands and feet, and the development of new hand tremors or worsening of an existing tremor
- Advise patients not to use the drug if they plan to become pregnant, or if their partner can become pregnant
Pregnancy Category:X
Breastfeeding: As per manufacturer's data, it is not known whether miglustat is excreted in breast milk. Because of the potential for serious adverse reactions in nursing infants from miglustat, a decision should be made whether to discontinue nursing or to discontinue the drug, analyzing the importance of the drug to the mother.
Drug Name: Zavesca 100 MG Oral Capsule
Ingredient(s): Miglustat
Imprint: OGT;918;100
Color(s): White
Shape: Capsule
Size (mm): 14.00
Score: 1
Inactive Ingredient(s): N/A
Drug Label Author:
Actelion Pharmaceuticals US, Inc.
DEA Schedule:
Non-Scheduled
