TREATMENT-EXPERIENCED* STUDIES

Female patient

*Treatment-experienced: patients previously treated with imiglucerase for a minimum of 30 consecutive months prior to switching to VPRIV2

†Patients aged ≥4 years currently being treated on a stable dose of imiglucerase for type 1 Gaucher disease may be switched to VPRIV at the last imiglucerase dose administered 2 weeks prior1

12-MONTH SWITCH: STUDY 034

STUDY 034 DESIGN (N=41)2

PATIENTS TREATED WITH IMIGLUCERASE
FOR ≥30 MONTHS

SWITCH
(no washout)

VPRIV
15–60 U/KG EOW (n=40)

PRIMARY OBJECTIVE

To evaluate the safety of every-other-week dosing of VPRIV in patients with type 1 Gaucher disease who were previously treated with imiglucerase.2

SECONDARY OBJECTIVES

Absolute change from baseline to 12 months in hemoglobin concentration; and percentage change from baseline to 12 months in platelet count, and spleen and liver volumes normalized by body weight.2

Over 12 months, stability vs. baseline was maintained in all four clinical parameters.2

HEMOGLOBIN CONCENTRATION

  • Median hemoglobin concentration at baseline: 13.8 g/dL2
  • Mean change at 12 months: −0.1 g/dL (within the pre-defined efficacy criterion of ±1 g/dL)2
Hemoglobin

PLATELET COUNT

  • Median platelet count at baseline: 162 × 109/L2
  • Mean change at 12 months: +7.0% (within the pre-defined efficacy criterion of ±20%)2
Platelets

SPLEEN VOLUME

  • Median spleen volume at baseline: 2.5 MN2
  • Mean change at 12 months: −5.6% (within the pre-defined efficacy criterion of ±15%)2
Damaged Spleen

LIVER VOLUME

  • Median liver volume at baseline: 0.8 MN2
  • Mean change at 12 months: 0.0% (within the pre-defined efficacy criterion of ±15%)2
Liver

MEAN CHANGE FROM BASELINE IN
HEMOGLOBIN CONCENTRATION2

12 months VPRIV 15–60 U/kg EOW

12-month data – median baseline: 13.8 g/dL; mean change from baseline:  −0.1 g/dL (within the pre-defined efficacy criterion of ±1 g/dL)2

MEAN % CHANGE FROM BASELINE IN
PLATELET COUNT2

12 months VPRIV 15–60 U/kg EOW

12-month data – median baseline: 162 × 109/L; mean change from baseline: +7.0% (within the pre-defined efficacy criterion of ±20%)2

MEAN % CHANGE FROM BASELINE IN
SPLEEN VOLUME2

12 months VPRIV 15–60 U/kg EOW

12-month data – median baseline: 2.5 MN; mean change from baseline: –5.6% (within the pre-defined efficacy criterion of ±15%)2

MEAN % CHANGE FROM BASELINE IN
LIVER VOLUME2

12 months VPRIV 15–60 U/kg EOW

12-month data – median baseline: 0.8 MN; mean change from baseline: 0.0% (within the pre-defined efficacy criterion of ±15%)2

EOW, every other week; MN,multiples of normal

VPRIV Clinical Program Overview
VPRIV Clinical Program Overview
VPRIV Safety & Tolerability Profile
VPRIV Safety & Tolerability Profile