FDA Approved Indications for this Orphan Drug:

Long-term enzyme replacement therapy for children, adolescents and adult patients with a confirmed diagnosis of Type I Gaucher disease who exhibit signs and symptoms that are severe enough to result in one or more of the following conditions: a) moderate-to-severe anemia; b) thrombocytopenia with bleeding tendency; c) bone disease; d) significant hepatomegaly or splenomegaly.

FDA Marketing Approval issued as of:

Orphan Drug exclusivity ends on:
Company Making and Marketing this drug:
Genzyme Corporation

FDA Designation Date:

FDA Drug Designation:
For replacement therapy in patients with Gaucher's disease type I.