BioMarin’s enzyme therapy granted six-month FDA review

RhASB (galsulfase) is BioMarin’s investigational enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI), also known as Maroteaux-Lamy Syndrome, 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). This enzyme deficiency leads to the accumulation of GAGs in the lysosomes of cells, giving rise to progressive cellular, tissue and organ system dysfunction.

Debilitating symptoms can include impaired cardiac and pulmonary function, delayed physical development, skeletal and joint deformities, impaired vision and hearing, sleep apnea, and reduced endurance. The majority of people with MPS VI die from disease-related complications between childhood and early adulthood.

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.

A six-month review is typically granted to drugs that, if approved, would be a significant improvement in the safety or effectiveness of the treatment, diagnosis, or prevention of a serious or life-threatening disease. The FDA previously granted rhASB orphan drug designation, a designation conferred upon investigational products for diseases that affect fewer 200,000 patients in the US.

The FDA will take action on the application by May 31, 2005.