Date: 2012-06-28
Type of information: Presentation of results at a congress
phase: 3
Announcement: results resented at the European Working Group on Gaucher Disease (EWGGD) meeting held in Paris, France, from June 28 – 30, 2012.
Company: Shire (UK-USA)
Product: Vpriv® (velaglucerase alfa)
Action
mechanism: This hydrolytic lysosomal glucocerebroside-specific enzyme is an enzyme replacement therapy.
Disease: Gaucher disease
Therapeutic area: Rare diseases
Country:
Trial details:
Latest
news: Shire has presented new data that show its enzyme replacement therapy for type 1 Gaucher,Vpriv® (velaglucerase alfa for injection), significantly improved selected markers of Gaucher-related bone disease in patients. These data were presented at the European Working Group on Gaucher Disease (EWGGD) meeting held in Paris, France, from June 28 – 30, 2012.
The data presented demonstrate that Vpriv® improves Gaucher-related bone disease by a sustained increase in bone mineral density (BMD). BMD refers to the measurement of mineral matter per square centimeter of bone measured by Z-scores. Z-scores allow for a comparison of a patient’s BMD to age- and sex-matched normalized scores in populations without Gaucher disease. In Gaucher disease patients, BMD is generally reduced compared to individuals without Gaucher disease, often resulting in lower Z-scores. Measuring BMD can help to quantify the impact of Gaucher disease on the patient’s bone and can help identify the potential benefits of treatment in improving Gaucher-related bone disease.
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 (LS) BMD in Gaucher patients starting at nine months of treatment with Vpriv® (P<0.05). Patients participating in the study were administered 60 U/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 60 U/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 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®.
