This indication concerns the use of tetrahydrobiopterin (6R-BH4) as a treatment for the endothelial dysfunction that causes vascular complications in diabetes, cardiovascular and other diseases. The newly acquired rights expand BioMarin’s market opportunity for 6R-BH4 well beyond phenylketonuria (PKU).
In November 2004, BioMarin announced two separate agreements with Daiichi Suntory Pharma relating to 6R-BH4 for genetic diseases including PKU: a license to intellectual property, preclinical and clinical data, and a manufacturing and supply agreement.
6R-BH4 is the active pharmaceutical ingredient in Phenoptin (sapropterin hydrochloride), BioMarin’s investigational phase III product candidate for the treatment of 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.