Inborn errors of metabolism:
Indications for ZAVESCA:
Mild to moderate type 1 Gaucher disease when enzyme replacement therapy is not feasible.
≥18yrs: 100mg 3 times daily; may reduce to 100mg once or twice daily if diarrhea or tremor occurs. Mild renal impairment (CrCl 50–70mL/min): 100mg twice daily. Moderate renal impairment (CrCl 30–50mL/min): 100mg once daily. Severe renal impairment: not recommended.
<18yrs: not established.
Perform neurological evaluations at baseline, then periodically at 6-month intervals. Reevaluate and consider discontinuation if signs/symptoms of peripheral neuropathy develop (eg, numbness, tingling). GI disease (eg, inflammatory bowel). Monitor platelets. Renal impairment. Elderly. Pregnancy. Nursing mothers: not recommended.
Glucosylceramide synthase inhibitor.
May antagonize imiglucerase.
Diarrhea, weight loss, tremor, flatulence, abdominal pain, GI disturbances, weakness, headache, dizziness, paresthesia, cramps, vision problems, thrombocytopenia, back pain, dry mouth, menstrual changes; reduced spermatogenesis, peripheral neuropathy.
Generic Drug Availability: