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Aldurazyme

BioMarin Pharmaceutical Inc.

Drug Names(s): laronidase, alpha-L-iduronidase (rhHP-IDUA)

Description: Aldurazyme (alpha-L-iduronidase) is a recombinant enzyme for the treatment of an inherited disease called mucopolysaccharidosis 1 (MPS 1). MPS 1 is characterized by a genetic mutation in an enzyme (endogenous alpha-L-iduronidase) that breakdowns certain sugars called mucopolysaccharides or glycosaminoglycans. This defect in enzyme function results in accumulation and storage of sugars/mucopolysaccharides in the lysosomal compartment. Aldurazyme, administered intravenously, breaksdown the accumulated sugars in the lysosome.

Deal Structure: In September 1998, BioMarin entered into a joint venture with Genzyme for the development and commercialization of Aldurazyme. Under the terms of the joint venture, Genzyme will be responsible for the marketing, distribution, sales and third party reimbursement of Aldurazyme. The companies share expenses and profits equally.

In January 2008, BioMarin and Genzyme announced a restructuring of their joint venture regarding Aldurazyme. Under the revised structure, the operational responsibilities for BioMarin and Genzyme will not significantly change. Genzyme will continue to globally market and sell Aldurazyme for mucopolysaccharidosis I and BioMarin will continue to manufacture Aldurazyme.

As of January 1, 2008, instead of sharing all costs and profits equally through the 50/50 joint venture, Genzyme will record sales of Aldurazyme and will pay BioMarin a tiered payment ranging from approximately 39.5 to 50 percent of worldwide net product sales, which will also be recorded by...See full deal structure in Biomedtracker

Partners: Sanofi


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