FOR YOUR TREATMENT-EXPERIENCED PATIENTS

Study 034: 12-Month Switch Study (n=41)1

Study 034: 12-MONTH SWITCH STUDY OBJECTIVES
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Patients treated with
imiglucerase for ≥30 months
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switch (no washout)
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VPRIV
15–60 U/kg EOW (n=40)
  • Primary: To evaluate the safety of every-other-week dosing of VPRIV in patients with type 1 Gaucher disease who were previously treated with imiglucerase.1

  • Secondary: 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.1

Over 12 months, stability vs. baseline was maintained in all four clinical parameters.1

mean change from baseline1

Up arrow 12 months VPRIV 15–60 U/kg EOW
Mean change from baseline
Hemoglobin
Hemoglobin
concentration
  • Median hemoglobin concentration at baseline: 13.8 g/dL1

  • Mean change at 12 months: −0.1 g/dL
    (within the pre-defined efficacy criterion of ±1 g/dL)1

mean change from baseline1

Up arrow 12 months VPRIV 15–60 U/kg EOW
Mean % change from baseline
Hemoglobin
Hemoglobin
concentration
  • Median hemoglobin concentration at baseline: 13.8 g/dL1

  • Mean change at 12 months: −0.1 g/dL
    (within the pre-defined efficacy criterion of ±1 g/dL)1

Platelets
Platelet count
  • Median platelet count at baseline: 162 × 109/L1

  • Mean change at 12 months: +7.0%
    (within the pre-defined efficacy criterion of ±20%)1

Spleen
Spleen volume
  • Median spleen volume at baseline: 2.5 MN1

  • Mean change at 12 months: −5.6%
    (within the pre-defined efficacy criterion of ±15%)1

Liver
Liver volume
  • Median liver volume at baseline: 0.8 MN1

  • Mean change at 12 months: 0.0%
    (within the pre-defined efficacy criterion of ±15%)1

EOW, every other week; MN, multiples of normal.

FOR YOUR TREATMENT-EXPERIENCED PATIENTS

Study 044: 5-Year Long-Term Extension Study (n=38)1

Study 044: 5-Year Long-Term Extension Study OBJECTIVES
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Study 034
Overall VPRIV (n=40)
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VPRIV 15–60 U/kg
EOW (n=38)
  • Primary: To evaluate the long-term safety of VPRIV treatment. 1

  • Secondary: To evaluate the effects of treatment on hemoglobin concentration, platelet count, and liver and spleen volumes.1

Patients previously treated with imiglucerase maintained stability vs. baseline
for up to 5 years after switching to VPRIV.1,2

MEAN INCREASE IN HEMOGLOBIN CONCENTRATION1

Hemoglobin
Mean change and 95% CI, g/dL

12-month data – baseline:* 13.8 g/dL; mean change from baseline: –0.1 g/dL
(within the pre-defined efficacy criterion of ±1 g/dL)3

24-month data – baseline:* 13.82 g/dL; mean change from baseline: –0.05 g/dL1,4

MEAN INCREASE IN PLATELET COUNT1

Platelets
Mean change and 95% CI, x109/L

12-month data – baseline:* 162 × 109/L; mean change from baseline: +7.0%
(within the pre-defined efficacy criterion of ±20%)3

24-month data – baseline:* 164.5 × 109/L; mean change from baseline: 9.03 × 109/L1,4

MEAN DECREASE IN SPLEEN VOLUME1

Spleen
Mean % change and 95% Cl

12-month data – baseline:* 2.5 MN; mean change from baseline: –5.6% (within the pre-defined efficacy criterion of ±15%)3

24-month data – baseline:* 0.821% of body weight; mean change from baseline: –0.110% of body weight1,4

MEAN DECREASE IN LIVER VOLUME1

Liver
Mean % change and 95% Cl

12-month data – baseline:* 0.8 MN; mean change from baseline: 0.0%
(within the pre-defined efficacy criterion of ±15%)3

24-month data – baseline:* 2.062% of body weight; mean change from baseline: –0.026% of body weight1,4

*Baseline is defined as before the first dose of VPRIV in Study 034

CI, confidence interval; MN, multiples of normal; EOW, every other week