VPRIV PATIENT POPULATION OVERVIEW
Patients can be on VPRIV during the various stages of life1,2
VPRIV has been evaluated during clinical trials in:
22
PEDIATRIC
PATIENTS
(4–17 years) in
Study 0443
73
ADULT
PATIENTS
(≥18 years) in
Study 0443
56
GERIATRIC
PATIENTS
(≥65 years, including 10 patients
>75 years) across all clinical studies4
Real-world experience data available in:
>300 PREGNANCIES4
Real-world data show the use of VPRIV was not associated with adverse outcomes in pregnancy.4,5*
These data have been reported in the pharmacovigilance database and published observational
cohort studies, including the International Collaborative Gaucher Group Registry.4,5
*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 outcomes4
VPRIV was evaluated across a diverse genotypic spectrum1
In clinical trials VPRIV was evaluated in patients with genotypic mutations associated with mild to severe disease.1,6
Population by GBA* genotype before first dose in initial trials1
| GBA GENOTYPE | Genotype Prevalence | OVERALL VELAGLUCERASE ALFA n=39 (%) |
IMIGLUCERASE-TO- VELAGLUCERASE ALFA n=16 (%) |
|---|---|---|---|
| N370S/N370S | Most common in the Ashkenazi Jewish and non-Jewish European population7 | 13 (33) | 2 (13) |
| N370S/84GG | Restricted to Ashkenazi Jewish population7 | 1 (3) | 0 |
| N370S/L444P | Prevalent worldwide but lower chances of developing neuropathic disease7,8 | 2 (5) | 1 (6) |
| L444P/L444P | Prevalent worldwide, associated with developing neuropathic disease7,8 | 2 (5) | 2 (13) |
| N370S/other | Most common in the Ashkenazi Jewish and non-Jewish European population7 | 13 (33) | 6 (38) |
| L444P/other | Prevalent worldwide7,8 | 3 (8) | 0 |
| F2131/F2131 | Relatively rare in Ashkenazi Jewish populations, higher prevalence in Japanese populations9 | 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.
