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