Phase 3 study of velaglucerase alfa for the treatment of type 1 Gaucher disease

Shire presented positive results from its first Phase 3 study (TKT032) of velaglucerase alfa for the treatment of Type 1 Gaucher disease. This trial was a 12-month, randomized, double-blind, parallel-group global study in 25 treatment-naive patients aged two years and older that evaluated velaglucerase alfa at dosages of 45 U/kg and 60 U/kg. The study's primary endpoint, mean hemoglobin concentration changes from baseline, was represented by a statistically significant increase of 23.3% (+2.43+/-0.32 g/dL, P< 0.0001) at 12 months in the 60 U/kg group. Secondary endpoints for both doses were changes in platelet counts, changes in organ volumes, changes in surrogate markers of Gaucher disease, and for the 45 U/kg dose only, change in hemoglobin concentrations from baseline.

Velaglucerase alfa is an investigational enzyme replacement therapy. Produced by Shire's gene-activation technology in a human cell line, the resulting enzyme product has the exact human amino acid sequence and a human glycosylation pattern.

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