BioMarin Pharmaceutical Inc. has announced that AnGes MG Inc, BioMarin's marketing and distribution partner in Japan, has submitted a Biologics License Application (BLA) for Naglazyme (galsulfase) to the Japanese Ministry of Health, Labour and Welfare.
"We are pleased to be working with AnGes to bring the first drug treatment option to MPS VI patients in Japan," said Stephen Aselage, senior vice president of global commercial development at BioMarin. "Patient advocacy groups and medical societies in Japan have shown a strong interest in Naglazyme, and we are honoured to bring this life-altering therapy to MPS VI patients in the US, Europe, and now to other parts of the world."
Naglazyme is the first and only enzyme replacement therapy indicated for the treatment of MPS VI. As the first drug approved for MPS VI, regulatory agencies in both the United States and European Union have granted Naglazyme orphan drug status, which confers seven years and 10 years of market exclusivity, respectively.
Naglazyme is indicated for patients with MPS VI. Naglazyme has been shown to improve walking and stair-climbing capacity.
BioMarin established a marketing and distribution agreement with AnGes in December 2006, through which AnGes obtained exclusive rights to market Naglazyme in the Japanese market. Naglazyme was approved by the US Food and Drug Administration (FDA) in May 2005 and by the European Commission (EC) in January 2006. As the first drug approved for MPS VI, the FDA and EC have both designated Naglazyme as an orphan drug, conferring seven years of market exclusivity in the United States and 10 years of market exclusivity in the European Union. In addition, Naglazyme obtained orphan designation in June 2007 from the Ministry of Health, Labour and Welfare (MHLW) in Japan.
MPS VI also known as Maroteaux-Lamy syndrome. It is a debilitating, life-threatening genetic disease caused by a deficiency of the enzyme N-acetylgalactosamine 4-sulfatase. This enzyme deficiency leads to the accumulation of certain complex carbohydrates, glycosaminoglycans (GAGs), in the lysosomes, giving rise to progressive cellular, tissue and organ system dysfunction. The majority of individuals with MPS VI die from disease-related complications between childhood and early adulthood.