Shire, a global specialty biopharmaceutical company based in Dublin, released new data that show VPRIV (velaglucerase alfa for injection), the company’s enzyme replacement therapy for type 1 Gaucher disease, significantly improved selected markers of Gaucher-related bone disease in patients.

The data demonstrate that VPRIV improves Gaucher-related bone disease by a sustained increase in bone mineral density (BMD)—the measurement of mineral matter per square centimeter of bone measured by Z-scores. In Gaucher disease patients, BMD is generally reduced compared to individuals without Gaucher disease, often resulting in lower Z-scores.

Results from a head-to-head phase III study (HGT-GCB-039) of VPRIV and Cerezyme, and follow-on extension trial (HGT-GCB-044) of VPRIV, demonstrate a statistically significant improvement in lumbar spine BMD in Gaucher patients starting at nine months of treatment with VPRIV (P<0.05). Patients participating in the study were administered 60U/kg every other week of either VPRIV or Cerezyme for nine months as part of the HGT-GCB-039 study. All patients, including those who received Cerezyme, subsequently received 60U/kg every other week of VPRIV for an additional 15 months in the extension trial (HGT-GCB-044).

Clinically and statistically significant improvement from baseline in mean lumbar spine 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 lumbar spine 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 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.

“These study results show that VPRIV is effective in treating selected markers of Gaucher-related bone disease, allowing these patients to achieve an important therapeutic goal quickly,” said Ari Zimran, Shaare Zedek Medical Center, Hebrew University and Hadassah Medical School, Jerusalem, Israel.