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 recommended.
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). Use effective methods of contraception during therapy; men should maintain contraception for 3 months after treatment. Elderly. Labor & delivery. Nursing mothers: not recommended.
Glucosylceramide synthase inhibitor.
May antagonize imiglucerase.
Diarrhea, weight loss, tremor, flatulence, abdominal pain, GI disturbances, weakness, headache, dizziness, paresthesia, cramps, thrombocytopenia, reduced spermatogenesis, peripheral neuropathy.