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 News Release - December 18, 2006

BioMarin Announces Positive Results From Phase 3 Extension Study of Phenoptin for PKU

Data Confirms Safety and Efficacy of Phenoptin in PKU Patients; On Track to File NDA in Second Quarter 2007

NOVATO, Calif., Dec. 18 (HSMN NewsFeed) -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN News) today announced positive results from the pivotal Phase 3 extension study of Phenoptin (sapropterin hydrochloride), an investigational oral small molecule for the treatment of patients with phenylketonuria (PKU), who have elevated phenylalanine (Phe) levels. Results confirm that all pre-specified safety and efficacy endpoints of the open-label extension study were met. Data demonstrated the long-term safety and tolerability of Phenoptin as a treatment to control blood Phe levels across a range of doses in PKU patients.

The 22-week multi-center open-label dose titration Phase 3 extension study enrolled 80 patients who had previously completed the pivotal Phase 3 clinical trial. Patients in the extension study received Phenoptin doses of 5 mg/kg/day, 10 mg/kg/day, and 20 mg/kg/day for two-week intervals each, followed by four weeks at 10 mg/kg/day. Patients completing this first phase of treatment were then treated for an additional 12 weeks with one of the three doses, depending upon their Phe levels at weeks two and six. A total of 79 patients completed both stages of treatment.

Key findings from the study:

-- Phenoptin demonstrated an excellent safety and tolerability profile for all three doses over the 22 weeks of treatment, which was the primary endpoint of the extension study. There were no withdrawals due to adverse events related to the study drug during the extension study.

-- Phenoptin provided a dose-dependent reduction in blood Phe levels relative to baseline, with average Phe level decreases of 100 umol/L (1.7 mg/dl), 204 umol/L (3.4 mg/dl), and 263 umol/L (4.4 mg/dl) for patients receiving doses of Phenoptin of 5 mg/kg/day, 10 mg/kg/day, and 20 mg/kg/day, respectively (secondary endpoint).

-- A once daily dose regimen of Phenoptin was sufficient to maintain the reduction of blood Phe levels throughout a 24 hour period.

-- The incidence and type of adverse events were comparable to that of the placebo group during the double-blind study and nearly all were mild or moderate in severity. The four most frequently reported events were headache, nasopharyngytis, vomiting, and diarrhea.

The ongoing Phase 3 diet study is designed to assess the increase in Phe tolerance levels in patients taking Phenoptin, and BioMarin remains on track to announce results from the Phase 3 diet study in the first quarter of 2007. BioMarin and Serono expect to file the NDA and MAA in the second and third quarters of 2007, respectively. In May 2005, BioMarin entered into a strategic partnership with Serono for the development and commercialization of Phenoptin for the treatment of PKU.

About Phenoptin

Phenoptin is an investigational oral small molecule therapeutic for the treatment of PKU. The active ingredient in Phenoptin, 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. Preliminary clinical data have suggested that Phenoptin has a potential to produce significant reductions in blood Phe levels in the subset of patients who are BH4- responsive. BioMarin and Serono estimate that Phenoptin could be a potential treatment option for approximately 30 percent to 50 percent of the estimated 50,000 individuals in the developed world who have been diagnosed with PKU.

Phenoptin received orphan drug designation to treat PKU from both the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMEA). If Phenoptin becomes the first drug therapy approved for the treatment of PKU, Phenoptin would 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 Phenoptin Fast Track designation, which is designed to facilitate the development and expedite the review 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 brain, resulting in a variety of complications including severe mental retardation and brain damage, mental illness, seizures and tremors, and cognitive problems. As a result of global newborn screening efforts implemented in the 1960s and early 1970s, virtually all PKU patients in developed countries have been diagnosed at birth. The only treatment currently available for PKU patients is a highly restrictive and expensive medical food diet that most patients fail to adhere to the extent needed for achieving adequate control of blood Phe levels. To learn more about PKU, please visit www.PKU.com. Information on this website is not incorporated by reference into this press release.

Positive Results from Phase 3 Clinical Study of Phenoptin for PKU

Positive results of a Phase 3, double-blind, placebo-controlled clinical study of Phenoptin (sapropterin dihydrochloride), an investigational oral small molecule for the treatment of phenylketonuria (PKU) were reported on March 15, 2006. Results confirmed that all pre-specified primary and secondary endpoints were met and data demonstrated a statistically significant reduction at six weeks in blood phenylalanine (Phe) levels (p<0.0001) in patients receiving Phenoptin, compared with those receiving placebo.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio is comprised of two approved products and multiple clinical and preclinical product candidates. Approved products include NaglazymeŽ (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin, and AldurazymeŽ (laronidase) for mucopolysaccharidosis I (MPS I), a product that BioMarin developed through a 50/50 joint venture with Genzyme Corporation. Investigational product candidates include Phenoptin(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 poorly controlled hypertension. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking Statements

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 Phenoptin; 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 Phenoptin; results and timing of current and planned clinical trials of Phenoptin for the treatment of PKU; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency and other regulatory authorities concerning Phenoptin; 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 2005 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.


Source: BioMarin Pharmaceutical

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