“BioMarin Pharmaceutical Inc. has announced positive results from the Phase 3 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. The data were presented at the annual meeting of the Society for Inherited Metabolic Diseases held in Pacific Grove, California.
Dr. Swiedler, senior vice president of clinical affairs at BioMarin, stated, “We are pleased to see continued benefit experienced by MPS VI patients receiving extended treatment with rhASB for MPS VI. The Phase 3 extension data further confirm the positive safety and efficacy profile of rhASB we have observed since the initiation of the clinical program, and we are working to make this therapy commercially available to individuals with MPS VI in the United States in the second half of the year.”
MPS VI (also known as Maroteaux-Lamy Syndrome) is a debilitating, life-threatening genetic disease for which no drug therapies are currently available. MPS VI is caused by a deficiency of the enzyme N-acetylgalactosamine 4-sulfatase, also known as arylsulfatase B. The deficiency leads to the accumulation of GAG in the lysosomes, the digestive organelles of the cell, giving rise to progressive cellular, tissue and organ system dysfunction.