Extension Study of rhASB in MPS VI Shows Improvements in Endurance

BioMarin Pharmaceutical has announced positive results from the Phase III extension study of rhASB (galsulfase), the company's investigational enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI).

Data from the study demonstrate that patients who received rhASB for an additional 24 weeks, for a total of 48 weeks, continued to experience improved endurance. Patients who initially received placebo and then received rhASB for 24 weeks also experienced improved endurance.

All 38 patients who completed the initial 24-week, multicenter, double-blind, placebo-controlled trial were enrolled in the open-label extension study to further evaluate the safety and efficacy of rhASB. Patients who received rhASB during the initial 24-week, placebo-controlled portion of the trial continued to receive weekly 1 mg/kg infusions of rhASB in the extension study.

Patients who initially received placebo during the placebo-controlled portion of the trial received 1 mg/kg of rhASB via weekly infusion during the extension study. Patients were evaluated at predefined, six-week intervals to assess changes in primary and secondary efficacy endpoints and the safety and tolerability of weekly rhASB infusions.