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Results From Investigator-Sponsored Trials Presented at SSIEM Conference

Wed, 09/01/2010 - 7:31am
Bio-Medicine.Org

NOVATO, Calif., Sept. 1 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that results from investigator-sponsored studies on Kuvan (sapropterin dihydrochloride) are being presented at the 2010 Annual Symposium of the Society for The Study of Inborn Errors of Metabolism (SSIEM), August 31st-September 3rd in Istanbul, Turkey. Investigators reported that PKU patients taking Kuvan showed improvements in brain functioning, mood and behaviors.

"While these investigator-sponsored studies were conducted in a small number of subjects, the results indicate benefits beyond lowering blood Phe levels, and we hope to document similar neurocognitive improvements in subjects with PKU in our recently initiated Phase 3b Kuvan outcomes study," said Dr. Hank Fuchs, Chief Medical Officer of BioMarin.  "If successful, the data will be submitted in support of a label amendment."

Highlights of Investigator-Sponsored Studies:Neurocognitive findings in individuals with Phenylketonuria and treatment with sapropterin dihydrochloride (BH4) (White, et al; Washington University, St. Louis): Baseline findings indicate that executive performance is worse for subjects with PKU as compared to non-PKU subjects across a range of executive functions such as inhibitory control, strategic processing and working memory. The results of this exploratory study suggest improvements in executive abilities following treatment with sapropterin.

Brain function in individuals with PKU treated with Kuvan: Evidence from functional magnetic resonance imaging (Christ, et al; University of Missouri-Columbia, USA): At baseline, irregularities in neural activation were observed in subjects with PKU in the prefrontal cortex (PFC) and other brain regions as compared to subjects without PKU. At four weeks, two early-treated PKU participants responded to Kuvan with

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