A Phase 2, Open-Label, Multi-Center Study Evaluating the Efficacy, Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 Patients

Gaucher disease is a genetic disease that results in a deficiency of an enzyme acid
beta-glucosidase, also known as glucocerebrosidase. This enzyme is needed to digest a
substrate (lipid) called glucosylceramide and, to a lesser degree, glucosylsphingosine. In
participants with Gaucher disease, the liver, spleen, bone marrow and brain show increases in
lipid concentration, specifically in cells derived from the monocyte/macrophage system.

Eliglustat tartrate (Genz-112638) is an oral drug that may regulate the Gaucher disease
process by decreasing the synthesis of glucosylceramide. The primary objective of this study
is to evaluate the efficacy, safety and pharmacokinetics (PK) of eliglustat tartrate,
administered as an oral dose of either 50 milligram (mg) twice daily (BID) or 100 mg BID, to
men and women with Gaucher disease Type 1 for 52 weeks.