Genzyme Study of Myozyme for Late-Onset Pompe Patients Meets Co-Primary Efficacy Endpoints
Genzyme Corp. announced that its Late Onset Treatment Study (LOTS) of Myozyme® (alglucosidase alfa) met its co-primary efficacy endpoints. The study was undertaken to evaluate the safety and efficacy of Myozyme in juvenile and adult patients with Pompe disease. Myozyme was first approved in 2006, and the product is now registered in 36 countries.
The randomized, double-blind, placebo-controlled study enrolled 90 patients at eight primary sites in the United States and Europe. Participants received either Myozyme or a placebo every other week for 18 months. The average age of study participants was 44 years. The primary efficacy endpoints of the study sought to determine the effect of Myozyme on functional endurance as measured by the six-minute walk test and to determine the effect of Myozyme on pulmonary function as measured by percent predicted forced vital capacity.
The results showed that, at 18 months, patients treated with Myozyme increased their distance walked in six minutes by an average of approximately 30 meters as compared with the placebo group (P=0.0283; Wilcoxon test). The placebo group did not show any improvement from baseline. The average baseline distance walked in six minutes in both groups was approximately 325 meters.
Percent predicted forced vital capacity in the group of patients treated with Myozyme increased by 1 percent at 18 months. In contrast, it declined by approximately 3 percent in the placebo group (P=0.0026; Wilcoxon test). The average baseline percent predicted forced vital capacity in both groups was approximately 55 percent.
The results for both efficacy endpoints were consistent across various prospectively defined subgroups.
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