GENZ submits Myozyme BLA

Genzyme (GENZ) reported Phase III results and submitted a BLA for Myozyme alglucosidase alfa enzyme replacement therapy to treat Pompe's disease. Myozyme met the primary endpoint of survival without invasive ventilator support at 18 months of age in the open-label, international Phase III AGLU01602 trial in 18 patients. At 18 months, 83% of Myozyme patients were both alive and free of ventilator support compared with 2% in a historical cohort. Outcomes were compared with a historical cohort due to the rapidly progressive and fatal nature of the disease. ...