Shire Reports Positive Results From First of Three Phase III Trials of velaglucerase alfa for Type 1 Gaucher Disease and Provides Important Updates on Interaction...(ty and efficacy of velaglucerase alfa in...),Health

Patients aged two years and older who were treatment naive were eligible to participate in the study if they presented with disease-related anemia and had at least one of the following clinical manifestations of Gaucher disease: thrombocytopenia, moderate splenomegaly or a readily palpable enlarged liver. Patients were randomized to receive velaglucerase alfa at either 45 U/ kg or 60 U/ kg for a duration of 12 months.

In the trial, the primary endpoint was reached with patients benefiting from a clinically important and statistically significant (p<0.0001) increase in mean hemoglobin concentration compared with baseline after receiving velaglucerase alfa at 60 U/kg IV every other week for 12 months. Statistically significant improvements compared with baselines were also observed in platelet and spleen sizes, and nominally significant improvements were observed in liver size at this dose. Results were clinically important as defined by standard criteria and consistent with the previously published Phase I/II data.

At the 45 U/kg IV dose, statistically significant improvements in hemoglobin, platelet count, and spleen volume were also demonstrated. The magnitude of changes in the 45U/kg dose was also clinically important, and a trend in liver volume reduction was observed. The 60U/kg dose performed numerically as well or better than 45U/kg across all measured clinical endpoints.

The specific data from this trial will be presented at a scientific meeting later this year.

Velaglucerase alfa was found to be generally well tolerated with no drug-related serious adverse events reported in the trial. No patients withdrew from the trial due to an adverse event.

Most of the drug-related adverse events were reported in association with velaglucerase alfa infusions, all of which were mild
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