No

Enzyme

Orphan drug: Treatment of Gaucher's disease

C

Hypersensitivity to any component

Prepared from pooled human placental tissue that may contain the causative agents of some viral diseases

>10%: Local: Discomfort, burning, and edema at the site of injection

<1%: Fever, chills, abdominal discomfort, nausea, vomiting

No obvious toxicity was detected after single doses of up to 234 units/kg

Refrigerate (4°C), do not shake

Glucocerebrosidase is an enzyme prepared from human placental tissue. Gaucher's disease is an inherited metabolic disorder caused by the defective activity of beta-glucosidase and the resultant accumulation of glucosyl ceramide laden macrophages in the liver, bone, and spleen; acts by replacing the missing enzyme associated with Gaucher's disease.

Half-life, elimination: ~4-20 minutes

Usually administered as a 20-60 unit/kg I.V. infusion given with a frequency ranging from 3 times/week to once every 2 weeks

None reported

None reported

No information available to require special precautions

No effects or complications reported

Treatment is required for life. Pregnancy/breast-feeding precautions: Inform prescriber if you are or intend to be pregnant. Consult prescriber if breast-feeding.

Parenteral: Dilute to a final volume of 100 mL or less of normal saline and infuse I.V. over 1-2 hours; an in-line filter should be used; do not shake solution as it denatures the enzyme

Monitor CBC, platelets, liver function tests

Injection: 10 units/mL (5 mL); 80 units/mL (5 mL)

Barton NW, Brady RO, Dambrosia JM, et al, "Replacement Therapy for Inherited Enzyme Deficiency - Macrophage-Targeted Glucocerebrosidase for Gaucher's Disease," N Engl J Med, 1991, 324(21):1464-70.

Whittington R and Goa KL, "Alglucerase: A Review of Its Therapeutic Use in Gaucher's Disease," Drugs, 1992, 44(1):72-93.