BioMarin marketing partner submits BLA in Japan for Naglazyme

US-headquartered BioMarin Pharmaceutical has said that its co-marketing and distribution partner in Japan, AnGes MG, has submitted a Biologics License Application (BLA) for the Maroteaux-Lamy syndrome (MPS VI) orphan drug treatment Naglazyme (galsulfase) to the Japanese Ministry of Health, Labour and Welfare.

MPS VI is a potentially fatal genetic disease caused by a deficiency of the enzyme N-acetylgalactosamine 4-sulfatase. A deficiency in the enzyme leads to the accumulation of certain complex carbohydrates in the cells of the body giving rise to progressive tissue and organ system damage and eventual failure. The majority of individuals with MPS VI die from disease-related complications between childhood and early adulthood.

BioMarin established a marketing and distribution agreement with AnGes in December 2006, where AnGes obtained exclusive rights to market Naglazyme on the Japanese market.

Naglazyme was approved by the 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, ensuring seven years of market exclusivity in the US and 10 years in the EU. The drug also obtained orphan designation in June 2007 from the Ministry of Health, Labour and Welfare (MHLW) in Japan.

Stephen Aselage, senior vice-president of global commercial development at BioMarin, said: "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, the EU and now to other parts of the world."

Q2 FY07 net sales of Naglazyme at BioMarin were USD 20.9m, compared with USD 18.4m for Q1 FY07, an increase of 13.6 per cent. Net sales for H1 FY07 were USD 39.3m, an increase of USD 22m on net sales of USD 17.3m for H1 FY06.