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

NOVATO, Calif., Sept. 27 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that a systematic literature review and meta-analysis of blood phenylalanine (Phe) levels and clinical outcomes in phenylketonuria (PKU) was published in the September/October 2007 issue of Molecular Genetics and Metabolism. The review of 40 studies with within-study correlations between blood Phe level and intelligence quotient (IQ) confirms a significant correlation between blood Phe level and IQ in PKU patients and supports the use of blood Phe levels as a predictive biomarker for IQ in clinical trials.

"This meta-analysis stresses the importance of early and continuous Phe control to prevent irreversible neurological damage in PKU patients and importantly, supports the use of blood Phe levels as the primary endpoint in our Kuvan clinical trials," said Emil Kakkis, M.D., Ph.D., Chief Medical Officer of BioMarin. "We are working closely with the FDA in the final months leading up to the PDUFA date and look forward to providing the first drug treatment option to PKU patients by the end of the year, pending FDA approval."

Highlights from the meta-analysis are summarized below:

-- Significant, proportional correlations were found during critical

periods (0 to 12 years of age) for early treated patients with PKU

(r=-0.35; 95% confidence interval), where each 100 umol/l increase in

blood Phe levels predicted an average 1.3 to 3.1 point reduction in IQ,

over a range of Phe from 423 umol/l to 750 umol/l.

-- Significant, proportional correlations were observed between IQ and

mean lifetime blood Phe level for early-treated patients

(r=34; 95% confidence interval), where each 100 umol/l increase in Phe

predicted a 1.9 to 4.1 point reduction in IQ, over a range of Phe from

394 umol/l to 666 umol/l.

-- These statistically significant correlations sugge
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SOURCE BioMarin Pharmaceutical Inc.
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