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Meta-Analysis of Blood Phe Levels and Clinical Outcomes in PKU Published

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 suggest that blood Phe

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


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 active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe mental retardation and brain damage, mental illness, seizures, tremors, and limited cognitive ability. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all PKU patients under the age of 40 in developed countries have been diagnosed at birth. Currently, PKU can only be managed by a Phe-restricted diet, which is supplemented by nutritional replacement products, like formulas and specially-manufactured foods; however, the strict diet is difficult for most patients to adhere to the extent needed for achieving adequate control of blood Phe levels. To learn more about PKU, please visit Information on this website is not incorporated by reference into this press release.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises two approved products and multiple clinical and preclinical product candidates. Approved products include Naglazyme(R) (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin, and Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation. Investigational product candidates include Kuvan(TM) (sapropterin dihydrochloride), a Phase 3 product candidate for the treatment of phenylketonuria (PKU), and 6R-BH4 for cardiovascular indications, which is currently in Phase 2 clinical development for the treatment of peripheral arterial disease and sickle cell disease. Both products are being developed in partnership with Merck Serono, a division of Merck KgA of Darmstadt, Germany. For additional information, please visit Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the development of its product candidate Kuvan; expectations regarding the Kuvan expanded access program; expectations regarding filings with regulatory agencies; and the development of 6R-BH4 for other indications. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: the results of ongoing clinical trials related to Kuvan; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency and other regulatory authorities concerning Kuvan; results and timing of current and planned clinical trials of 6R-BH4 for other indications; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's 2006 Annual Report on Form 10-K, as amended, and the factors contained in BioMarin's reports on Form 8-K. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.


Investors Media

Eugenia Shen Susan Berg

BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc.

(415) 506-6570 (415) 506-6594

SOURCE BioMarin Pharmaceutical Inc.
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