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BioMarin Pharmaceutical Inc.

Drug Names(s): rhASB, arylsulfatase B, Aryplase, galsulfase

Description: Aryplase (recombinant human arylsulphatase B, or rhASB) is being developed for Mucopolysaccharidoses VI or MPS VI, a lysosomal storage disease. MPS VI is characterized by an inherited deficiency in an enzyme, N-Acetylgalactosamine 4-sulfatase, required for the breakdown certain complex sugars called mucopolysaccharides or glycosaminoglycans (GAGs). The deficiency leads to accumulation of GAGs in the lysosome. Aryplase is administered intravenously for the life of the patient. Aryplase is BioMarin’s second enzyme replacement therapy product after Aldurazyme for MPS I, which is on the market.Phase II clinical trails with Aryplase are complete. Patient enrollment for a placebo controlled Phase III trials were completed in October 2003 and results are expected in second quarter of 2004.

Deal Structure: Revenue splits for this drug are BioMedTracker estimates.

AnGes and BioMarin
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. AnGes paid an upfront payment and will make milestone payments to BioMarin.

BioMarin and SA Pathology
In November 2011, BioMarin Pharmaceutical announced that it completed the buy back of certain intellectual property from SA Pathology, a unit of the Central Adelaide Local Health Network, for an upfront payment of $81 million. The intellectual property includes patents related to the purified form of Naglazyme and the method of using the enzyme in the treatment of MPS VI. Prior to this transaction, BioMarin licensed this intellectual property from SA Pathology and paid a five percent royalty on net sales of Naglazyme.

Partners: AnGes MG

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