Shire’s Gaucher Drug Meets Endpoints

Shire achieved positive results from its first Phase III study (TKT032) evaluating the safety and efficacy of velaglucerase alfa, an investigational enzyme replacement therapy for the treatment of Type 1 Gaucher disease. Data from a pediatric study and five-year follow-up results from a long-term Phase I/II extension study (TKT025 EXT) in adults also support long-term safety and efficacy of velaglucerase alfa in patients with Type 1 Gaucher disease. Both studies (TKT032 and TKT025 EXT) met their primary end-points.

TKT032 was a 12-month, randomized, double-blind, parallel-group global study in 25 treatment-naive patients aged two and older that evaluated velaglucerase alfa at 45 U/kg and velaglucerase alfa at 60 U/kg. The primary endpoint was change in hemoglobin concentrations from baseline at 60 U/kg. 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.

In the 60 U/kg group, statistically significant changes in mean hemoglobin concentration increased 23%; mean platelet count increased 66%; and mean spleen volume decreased 50%. Additionally, mean liver volume decreased 17%.