Clinically and statistically significant improvement from baseline in mean LS Z-score was seen at nine months of treatment with VPRIV, but not in the cohort of patients treated with Cerezyme. BMD, evaluated as an exploratory endpoint in the Phase III and extension studies, was measured by dual-energy x-ray absorptiometry (DEXA scan). Median LS Z-scores at baseline were -1.46 (-3.50, 0.98) in patients treated with VPRIV, and -0.86 (-2.17, 2.02) in patients treated with Cerezyme. Mean changes from baseline in LS Z-scores at nine months were 0.33 (0.10, 0.55) and 0.06 (-0.22, 0.34), respectively. Following an additional 15 months of treatment, mean change in LS Z-scores improved to 0.64 (0.22, 1.06) for patients initially treated with VPRIV and improved to 0.54 (0.21, 0.87) for patients who switched to VPRIV from Cerezyme at nine months. Femoral neck changes from baseline in both cohorts were non-significant (P>0.05) at either nine or 24 months. Analysis presented at EWGGD excluded data from five patients on concomitant bisphosphonates, although similar results were observed when data from these patients were included. The safety events observed in this study were similar to those seen historically in patients treated with VPRIV.
Shire presented additional data showing how VPRIV significantly improved Gaucher-related bone disease in patients at EWGGD:
- Bone Parameters in Adults with Type 1 Gaucher Disease Treated with Velaglucerase Alfa in Trial TKT025 and the Extension Study: Focus on the Bone Marrow Burden Scores Over 7 Years: encore presentation
- Bone Mineral Density in Adults with Type 1 Gaucher Disease Receiving Velaglucerase Alfa 60 U/kg Every Other Week: 2-Year Results (HGT-GCB-032/039/044)
VPRIV is made in a human cell line using Shire's gene activation technology. The enzyme produced has the exact human amino acid sequence as that found in the naturally occurring human enzyme.