Brand and Other Names:Zavesca
- Classes: Enzyme Inhibitors
Dosing & Uses
Dosage Forms & Strengths
Indicated as monotherapy for treatment of adults with mild-to-moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option
100 mg PO q8hr; decrease to qDay or q12hr for patients with adverse effects
Neimann-Pick Disease (Orphan)
Treatment of the neurological manifestations of Niemann-Pick disease type C
Orphan indication sponsor
- Amicus Therapeutics, Inc; 6 Cedar Brook Drive; Cranbury, NJ 08512
Mild (CrCl 50-70 mL/min/1.73 m²): 100 mg PO q12hr
Moderate (CrCl 30-50 mL/min/1.73m²): 100 mg PO qDay
Severe (CrCl <30 mL/min/1.73m²): Not recommended
Dosage Forms & Strengths
Safety and efficacy not established
Niemann-Pick Type C Disease (Off-Label)
Approved in Europe for NP-C in children >4 years
Limited data suggests a dose of 200 mg PO q8hr; adjust for body surface area with young children as follows
BSA >1.25 m²: 200 mg PO q8hr
BSA >0.88 - 1.25 m²: 200 mg PO q12hr
BSA >0.73-0.88 m²: 100 mg PO q8hr
BSA >0.47-0.73 m²: 100 mg PO q12hr
BSA ≤0.47 m²: 100 mg PO qDay
Monitoring: Reduced growth may occur in children because of diarrhea and weight loss; monitor during treatment
For elderly, dose with caution at low end of dosing range
Serious - Use Alternative
Significant - Monitor Closely
Weight loss (66-70%)
Abdominal pain (18-67%)
Visual disturbances (17%)
Memory impairment (8%)
Abdominal distension (8%)
Epigastric pain (<6%)
Hypersensitivity to product or components
Cases of peripheral neuropathy have been reported; patients should undergo baseline & repeat neuro evaluation at 6 month intervals; consider treatment cessation if symptoms develop
Tremor may develop or be exacerbated; dose reduction may ameliorate, usually within days; discontinuation may be required
Avoid high carbohydrate foods during treatment to lessen side effect of diarrhea; weight loss reported due to the diarrhea; incidence decreases over time
May affect spermatogenesis & sperm parameters; male patients should use reliable contraception
May increase clearance of Cerezyme (imiglucerase); concomitant use not recommended
MIld decrease in platelet counts reported; bleeding not reported
Exacerbation of existing tremor may occur that may resolve over time or improve with dose reduction; may consider discontinuing therapy if it does not resolve
Pregnancy & Lactation
Pregnancy Category: X
Lactation: Unknown; not recommended; discontinue drug or do not nurse
A:Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.
B:May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk.
C:Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done.
D:Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk.
X:Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist.
NA:Information not available.
Mechanism of Action
Competitive and reversible inhibitor of glucosylceramide synthase, the initial enzyme in a series of reactions resulting in synthesis of most glygosphingolipids; decreases rate of glycosphingolipid glucosylceramide formation
Half-Life: 6-7 hr
Peak Plasma Time: 2-2.5 hr
Protein Bound: Does not bind to plasma proteins
Vd: 83-105 L
Metabolism: Not metabolized
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