The US Food and Drug Administration (FDA) has accepted for filing and assigned six-month review to BioMarin Pharmaceutical's Biologics License Application (BLA) for rhASB (galsulfase), the company's investigational enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI). The FDA will take action on the application, under the Prescription Drug User Fee Act (PDUFA), by May 31, 2005.
The FDA previously granted rhASB orphan drug designation, a designation conferred upon investigational products for diseases that affect fewer 200,000 patients in the United States. Products with orphan drug designation that are the first to be approved for a specific indication have seven years market exclusivity within the United States.

rhASB is an investigational enzyme replacement therapy designed to address the underlying enzyme deficiency associated with MPS VI. If approved, rhASB could become the first drug therapy for the treatment of MPS VI.

MPS VI, also known as Maroteaux-Lamy Syndrome, is an inherited debilitating, life-threatening disease which affects approximately 1,100 people worldwide. MPS VI is caused by the deficiency of N-acetylgalactosamine 4-sulfatase (arylsulfatase B), a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). The majority of people with MPS VI die from disease-related complications between childhood and early adulthood.