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VPRIV PATIENT POPULATION

Male patient

VPRIV has been evaluated during clinical trials in:

24

PEDIATRIC
PATIENTS

(4–17 years) in Study 0444

73

ADULT
PATIENTS

(≥18 years) in Study 0445

56

GERIATRIC
PATIENTS

(≥65 years, including 10 patients
≥75 years) across all clinical studies1

Real-world experience data available in:

300+

PREGNANCIES

Real-world data for VPRIV in over 300 pregnancies have not identified an association with major birth defects, miscarriage, or adverse maternal or fetal outcomes.*1,6

Available data cannot definitively establish or exclude the absence of a VPRIV-associated risk
during pregnancy.1

*While available data cannot definitively establish or exclude the absence of a VPRIV-associated risk during pregnancy, these data have not identified an association with the use of VPRIV during pregnancy and major birth defects, miscarriage, or adverse maternal or fetal outcomes1,6

These data have been reported in the pharmacovigilance database and published observational cohort studies, including the International Collaborative Gaucher Group Registry.1,6

VPRIV was evaluated across a diverse genotypic spectrum7.

In clinical trials, VPRIV was evaluated in patients with genotypic mutations associated with mild to severe disease.7,8

Population by GBA† genotype before first dose in initial trials.7

GBA GENOTYPE GENOTYPE PREVALENCE OVERALL VPRIV
N=39 (%) 		IMIGLUCERASE-
TO-VPRIV
N=16 (%)
Asn409Ser/Asn409Ser Most common in the Ashkenazi Jewish and non-Jewish European population9,10 13 (33) 2 (13)
Asn409Ser/c.84dupG Restricted to the Ashkenazi Jewish population9,10 1 (3) 0
Asn409Ser/Leu483Pro Prevalent worldwide but lower chances of developing neuropathic disease9–11 2 (5) 1 (6)
Leu483Pro/Leu483Pro Prevalent worldwide, associated with developing neuropathic disease9–11 2 (5) 2 (13)
Asn409Ser/other Most common in the Ashkenazi Jewish and non-Jewish European population9,10 13 (33) 6 (38)
Leu483Pro/other Prevalent worldwide9-11 3 (8) 0
F2131/F2131 Relatively rare in Ashkenazi Jewish populations, higher prevalence in Japanese populations12 0 2 (13)
Other/other N/A 5 (13) 3 (19)

†The GBA gene is responsible for the production of the glucocerebrosidase enzyme
GBA, glucosylceramidase beta

VPRIV Clinical Program Overview
VPRIV Clinical Program Overview
How ERT Works
VPRIV Safety & Tolerability Profile