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TREATMENT-NAÏVE* STUDIES

VPRIV is a first-line option for your treatment-naïve patients1,2

The recommended starting dose for treatment-naïve patients 4 years of age and older is 60 U/kg once every other week as a 60-minute IV infusion.1 VPRIV should be administered under the supervision of a healthcare professional.1

male VPRIV patient hero blob

*Treatment-naïve: all patients were naïve to ERT at the start of the core studies, although they received different ERTs for the first 9 months3

STUDY 044 DESIGN (N=57)3

STUDIES 032 & 039 OVERALL VPRIV
(n=41)
STUDY 039 IMIGLUCERASE
TO VPRIV
(n=16)
VPRIV
60 U/KG EOW

(N=57)

PRIMARY OBJECTIVE

To evaluate the long-term safety of VPRIV treatment.3

SECONDARY OBJECTIVES

To evaluate the effects of treatment on hemoglobin concentration, platelet count, and liver and spleen volumes.3

One patient from each group in Study 039 did not participate in Study 044

At 24 months of VPRIV treatment, patients demonstrated improvements from baseline in clinical parameters. Improvements were maintained for up to 5 years.1,3

Increase

MEAN INCREASE IN HEMOGLOBIN
CONCENTRATION3

12-month data – baseline: 11.0 g/dL;
mean change from baseline: +2.2 g/dL ± 0.2 (SE)4
24-month data – mean change from baseline: +2.8 g/dL3,4

Increase

MEAN INCREASE IN PLATELET COUNT3

12-month data – baseline: 108.6 × 109/L;
mean change from baseline: +78 × 109/L ± 12 (SE)4
24-month data – mean change from baseline: +87.9 × 109/L4

Decrease

MEAN DECREASE IN SPLEEN VOLUME3

12-month data – baseline: 3.8% of body weight;
mean change from baseline: −2.1% ± 0.3 of body weight (SE)4
24-month data – mean change from baseline: −2.7% of body weight4

Decrease

MEAN DECREASE IN LIVER VOLUME3

12-month data – baseline: 4.0% of body weight;
mean change from baseline: −0.79% ± 0.15 of body weight (SE)4
24-month data – mean change from baseline −1.2% of body weight4

Baseline is defined as before the first dose of VPRIV in Studies 032 and 039

BL, baseline; EOW, every other week; SE, standard error

VPRIV’s safety and efficacy profile was similar in pediatric (ages 4–17 years) and adult patients.1

    In a subset of 24 pediatric patients from Study 044, safety results were consistent with those of the overall population (N=95):

  • No new safety concerns were identified5
  • No serious adverse events were related to treatment with VPRIV5
  • No serious infusion-related adverse events occurred5
  • No patient discontinued the study due to an adverse event5
  • One pediatric patient tested positive for IgG anti-velaglucerase antibodies in Study 0445

    – No apparent impact was noted on the efficacy of VPRIV as assessed by changes in hemoglobin concentration and platelet count5

  • Adverse reactions more commonly seen in pediatric patients compared to adult patients include (>10% difference): rash, aPTT prolonged, and pyrexia1
  • Safety and efficacy have not been established in children younger than 4 years1

Mean % change in 24 months in treatment-naïve pediatric patients

HEMOGLOBIN CONCENTRATION

Median hemoglobin concentration at baseline§: 10.75 g/dL5
Mean change at 24 months: +2.65 g/dL5

Hemoglobin

+24.3%
(n=8)5

Arrow

PLATELET COUNT

Median platelet count at baseline§: 116 × 109/L5
Mean change at 24 months: +79.13 × 109/L5

Platelet

+93.4%
(n=8)5

Arrow
Arrow

−66.3%
(n=7)5

Damaged Spleen

SPLEEN VOLUME

Median spleen volume at baseline§: 14.3 MN5
Mean change at 24 months: −2.55% of body weight5

Arrow

−27.5%
(n=8)5

Liver

LIVER VOLUME

Median liver volume at baseline§: 1.6 MN5
Mean change at 24 months: −1.14% of body weight5

−100

−80

−60

−40

−20

0

20

40

60

80

100

§Baseline is defined as before the first dose of VPRIV in Studies 032 and 039 MN, multiples of normal

Patients meeting pre-defined clinical parameters at 4 years.6

STUDY DESIGN

A post hoc analysis was conducted on 39 treatment-naïve patients, aged 6–62 years, from the Study 032 and 039 populations who enrolled in the 044 extension trial. This analysis assessed achievement of published pre-defined clinical parameters at baseline and yearly intervals after initiation of VPRIV for up to 4 years6

PRIMARY OBJECTIVE: To measure the number of patients who met these pre-defined clinical parameter expectations in hemoglobin and platelet concentration, and spleen and liver volumes6

STUDY LIMITATIONS

Benefits of achieving clinical parameters for a patient’s long-term clinical outcomes have not been demonstrated, and composite analyses may be required to assess the effect of ERT on Gaucher disease, instead of individual assessments6

This analysis does not cover the primary or secondary objectives of the 044 extension trial (Hughes et al. publication). This was a post hoc analysis.

These are observational data, therefore, cautious interpretation is advised. The clinical significance of these data is unknown. There are no official treatment guidelines for type 1 Gaucher disease, therefore, clinical parameters were pre-defined by Pastores et al., based on medical literature data and the clinical experience of an international panel of physicians.6

Pre-defined clinical parameters:

Parameters met by:

HEMOGLOBIN CONCENTRATION6

  • ≥11 g/L for females or patients 12 years and younger
  • ≥12 g/dL for males or patients older than 12 years

19/20 patients (95%)

Hemoglobin

PLATELET COUNT6

  • ≥100% increase if baseline is <60 × 109/L
  • ≥100 × 109/L if baseline is ≥60 and <120 × 109/L
  • ≥120 × 109/L if baseline is ≥120 × 109/L

20/20 patients (100%)

Platelet

SPLEEN VOLUME6

  • ≥50% decrease compared with baseline (or ≤8.0 MN)

16/16 patients (100%)

Damaged Spleen

LIVER VOLUME6

  • ≥30% decrease compared with baseline (or ≤1.5 MN)

15/16 patients (94%)

Liver
MN, multiples of normal

Over the 4 years, pre-defined clinical parameters were also maintained by 100% of patients who had already met these at baseline6

IMPORTANT SAFETY INFORMATION

Hypersensitivity reactions, including anaphylaxis, have occurred. The most serious adverse reactions in patients treated with VPRIV were hypersensitivity reactions.

Hypersensitivity reactions were the most commonly observed adverse reactions in patients treated with VPRIV in clinical studies. Patients were not routinely pre-medicated prior to infusion of VPRIV.