Zavesca: Patients Switched from ERT

Switched Patients Study Design*

6-month open-label randomized active-controlled study
36 adults with type 1 Gaucher disease who had been receiving ERT with Cerezyme®† (imiglucerase) for a minimum of 2 years
33 patients completed the study
Patients were randomized to 1 of 3 arms:
— Cerezyme at patient’s regular dose at baseline
— Zavesca 100 mg tid
— Cerezyme at patient’s regular dose at baseline + Zavesca 100 mg tid

Zavesca Maintained Benefits of Long-Term ERT in Organ Volume

Patients on ERT for a mean of 5.6 years
Liver and spleen volume were maintained after 6 months of Zavesca monotherapy1,2

Mean baseline values1,2:
Cerezyme group: 1.81 L
Zavesca group: 1.58 L
Combination group: 2.01 L
Baseline range: 1.2 to 1.3 x normal
aSignificant decrease in liver volume compared to the Cerezyme study group

Mean baseline values1,2:
Cerezyme group: 0.61 L
Zavesca group: 0.69 L
Combination group: 0.76 L
Baseline range: 5.3 to 5.5 x normal

Zavesca Maintained Hematological Benefits of
Long-Term ERT

Patients on ERT for a mean of 5.6 years
Hemoglobin concentration and platelet count were maintained after 6 months of Zavesca monotherapy1,2

Mean baseline values^1,2:
Cerezyme group: 13.18 g/dL
Zavesca group: 12.44 g/dL
Combination group: 12.38 g/dL
6 (17%) anemic at baseline
Anemia defined as hemoglobin concentration <11.5 g/dL

Mean baseline values^1,2:
Cerezyme group: 165.75 x 109/L
Zavesca group: 170.55 x 109/L
Combination group: 152.14 x 109/L
16 (48%) thrombocytopenic at baseline
Thrombocytopenia defined as platelet
count <150 x 109/L
bSignificant decline in platelet count compared to the Cerezyme study group

*This study is referred to as “Study 3” in the Zavesca package insert.
†Cerezyme is a registered trademark of Genzyme Corporation.
‡All p values are changes from baseline.

1. Zavesca® (miglustat) package insert. Actelion Ltd. 2003.
2. Data on file, Actelion Pharmaceuticals US, Inc.