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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