June 28, 2012
Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, today presented 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. These data were presented at the European Working Group on Gaucher Disease (EWGGD) meeting held in
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.
"Many type 1 Gaucher disease patients experience bone abnormalities," said Professor
, Shaare Zedek Medical Center,
and Hadassah Medical School,
. "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."
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).