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Meta-Analysis of Blood Phe Levels and Clinical Outcomes in PKU Published
Date:9/27/2007

st that blood Phe

level can be used reliably as a predictive biomarker for IQ in clinical

trials.

Kuvan(TM) (sapropterin dihydrochloride), an investigational oral small molecule for the treatment of PKU, is being developed in partnership with Merck Serono, a division of Merck KGaA, Darmstadt, Germany.

About Kuvan

Kuvan is an investigational oral small molecule therapeutic for the treatment of PKU. The active ingredient in Kuvan, sapropterin dihydrochloride, is the synthetic form of 6R-BH4 (tetrahydrobiopterin), a naturally occurring enzyme cofactor that works in conjunction with phenylalanine hydroxylase (PAH) to metabolize Phe. Clinical data suggest that treatment with Kuvan results in significant reductions in blood Phe levels in BH4-responsive patients. It also may enable some patients to minimize or eliminate highly-restrictive dietary constraints by increasing Phe tolerance levels. BioMarin and Merck Serono estimate that Kuvan could be a potential treatment option for approximately 30 percent to 50 percent of the estimated 50,000 identified PKU patients in the developed world.

Kuvan has received orphan drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMEA). If approved, it will receive seven years of market exclusivity in the United States and 10 years in the European Union for this indication. Additionally, the FDA has granted Kuvan Fast Track designation, which is designed to facilitate the development of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.

About PKU

PKU, a genetic disorder affecting approximately 50,000 diagnosed patients in the developed world, is caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). PAH is required for the metabolism of phenylalanine (Phe), an essential amino acid found in most protein-containing foods. If the
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SOURCE BioMarin Pharmaceutical Inc.
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