BioMarin Pharmaceutical Inc. announced that Japan's Ministry of Health, Labor, and Welfare (MHLW) 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.
"I am very pleased that enzyme replacement therapy with Aldurazyme has finally been approved in Japan for the treatment of MPS I," stated Yoshikatsu Eto, M.D., chairman and Professor in the Department of Pediatrics at Tokyo Jikei University Hospital located in Tokyo, Japan. "This is an important milestone for Japanese MPS I patients and their treating physicians who now have an effective treatment for this debilitating and life-threatening disease."
Aldurazyme was developed in partnership between BioMarin and Genzyme and was approved in the United States and the European Union in April and May 2003, respectively. Pursuant to the joint venture, BioMarin is responsible for manufacturing Aldurazyme and Genzyme is responsible for its commercialization. All expenses and profits and are shared equally between the companies.
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. Resulting symptoms, which span a spectrum of severity, can include impaired cardiac and pulmonary function, delayed physical development, skeletal and joint deformities, reduced endurance, and in some cases, delayed mental function. A majority of patients die before adulthood from complications of the disease.
Aldurazyme (laronidase) is indicated in the United States for patients with Hurler and Hurler-Scheie forms of MPS I and for patients with the Scheie form who have moderate to severe symptoms. The risks and benefits of treating mildly affected patients with the Scheie form have not been established. Aldurazyme has been shown to improve pulmonary function and walking capacity. Aldurazyme has not been evaluated for effects on central nervous system manifestations of the disorder.