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BioMarin Pharmaceutical Inc.

Drug Names(s): sapropterin dihydrochloride, 6R-BH4, BH4

Description: This drug is the active ingredient of Phenopterin, Biomarin's synthetic version of the naturally occurring enzyme cofactor Tetrahydrobiopterin (6R-BH4), but at a different dose. In addition to playing a role in the metabolism of a number of compounds (phenylalanine, tryptophan, tyrosine, and alkylglycerol), tetrahydrobiopterin is a cofactor for the enzyme nitric oxide synthase, which is involved in the production of nitric oxide (NO).

NO is a hormone-like molecule that regulates dilation and constriction of blood vessels. It has been suggested that in diseases such as diabetes, hypertension, and atherosclerosis, there is a BH4 deficiency that correlates with decreased NO synthesis and with endothelial dysfunction.

Deal Structure: In November 2004, BioMarin announced two separate agreements with Daiichi Suntory Pharma relating to 6R-BH4 for genetic diseases including PKU. The new license extends BioMarin's exclusive rights to Daiichi Suntory Pharma's patents and data related to 6R-BH4 for all indications. In exchange for the exclusive rights to the intellectual property and data for all indications, in all territories outside of Japan, BioMarin will pay Daiichi Suntory Pharma an upfront payment, an undisclosed royalty on sales of 6R-BH4, and developmental milestones for up to two indications.

In May 2005, BioMarin licensed from DaiichiSuntory Pharma Co., Ltd. the exclusive worldwide rights (excluding Japan) for the use of tetrahydrobiopterin (6R-BH4). These rights are covered by U.S., European and other foreignpatents and patent applications.

Also in May 2005, the Company entered into an agreement with Serono S.A. for the further development and commercialization of Phenoptin and Phenylase for PKU, and...See full deal structure in Biomedtracker

Partners: Daiichi Sankyo Co., Ltd. Merck KGaA

Tetrahydrobiopterin News

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