BIOMARIN WINS JAPANESE APPROVAL FOR ALDURAZYME

BioMarin Pharmaceutical has announced that Japan's Ministry of Health, Labor and Welfare has granted marketing authorization for Aldurazyme (laronidase), the first specific treatment approved in Japan for patients with the genetic disease mucopolysaccharidosis I (MPS I). Aldurazyme was approved in Japan as an orphan drug.

Aldurazyme was developed under a partnership between BioMarin and Genzyme and was approved in the United States and the European Union in 2003. Under the joint venture, BioMarin is responsible for manufacturing Aldurazyme, and Genzyme is responsible for its commercialization.

MPS I is a progressive, debilitating and fatal genetic disease caused by a deficiency of the enzyme alpha-L-iduronidase. This deficiency leads to the accumulation of complex carbohydrates in the lysosomes of cells, leading to the progressive dysfunction of cellular, tissue and organ systems. A majority of patients die before adulthood from complications of the disease, according to BioMarin. Aldurazyme has been shown to improve pulmonary function and walking capacity. The drug has not been evaluated for effects on the CNS manifestations of the disorder.