Genzyme Reports Interim Results From Pivotal Study of Myozyme

Genzyme has completed a planned analysis of interim data from its pivotal clinical trial of Myozyme, which is being studied for the treatment of Pompe disease.

The interim analysis was included in the trial's protocol to allow for the potential expedited submission of a biologic license application. It found that the trial has already met one of its key secondary efficacy endpoints and that there is a high probability the study will meet its primary efficacy endpoint upon completion.

The pivotal trial, known as AGLU01602, includes 18 patients with infantile-onset Pompe disease. These patients were enrolled in the trial and began receiving Myozyme (alglucosidase alfa) by 6 months of age. Because of the rapidly progressive and fatal nature of infantile-onset Pompe disease, outcomes for these patients are being compared with a matched historical cohort rather than a placebo cohort.

The study's primary endpoint is the proportion of patients treated with Myozyme who are alive and free of invasive ventilator support at 18 months of age, compared with the proportion of patients who were alive at 18 months of age in the historical cohort (2 percent). Results for the primary endpoint will be known this summer, when patients will have completed 52 weeks of treatment.