BioMarin, Genzyme to file MPS-1 therapy

BioMarin Pharmaceutical and partner Genzyme General have completed aPhase III clinical trial of Aldurazyme (laronidase), an investigational enzyme replacement therapy for patients with the life-threatening genetic disease mucopolysaccharidosis-1, and say they plan to meet with US, Canadian and European regulatory authorities to discuss applications to market the drug.

The trial enrolled 45 patients who were randomized to receive weekly intravenous infusions of Aldurazyme or placebo during a 26-week evaluation period. The 45 patients, with an average age of 15, have elected to receive Aldurazyme in an open-label extension study. The safety profile was comparable between the treatment and placebo groups, and there were no Aldurazyme-related serious adverse events.

A preliminary data analysis reveals a statistically significant increase in pulmonary capacity (p=0.028), and a positive trend in endurance as measured by a six-minute walk test (p=0.066), for Aldurazyme versus placebo. Among other endpoints measured in the trial, the main findings of an earlier open-label study of Aldurazyme were confirmed, including a reduction in liver size and a reduction in excretion of urinary glycosaminoglycans, the carbohydrate substances that accumulate in patients with MPS-1.