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BioMarin Announces Positive Opinion for Naglazyme From European Regulatory Authority
Final Approval Anticipated in Early 2006
Sep 15, 2005
NOVATO, Calif.

BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Evaluation Agency (EMEA), has issued a positive opinion on the company's Marketing Authorization Application for Naglazyme(TM) (galsulfase) an investigational enzyme replacement therapy for treatment of mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome).

The committee's proposed label states that Naglazyme is approved in the European Union as a long-term enzyme replacement therapy in patients with a confirmed diagnosis of MPS VI to treat the clinical manifestations of the disease. All clinical post-authorization commitments requested by the CHMP will be fulfilled through a clinical surveillance program that will monitor patients on commercial therapy. No additional clinical trials are required.

"We are pleased that the CHMP has recommended approval of Naglazyme for MPS VI and look forward to receiving a final determination from the European Commission in the next three to four months," stated Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "Our effort to establish a small, Europe-based sales force to market Naglazyme is well under way and, pending final approval from the European Commission, we expect to initiate product launch in early 2006."

The positive opinion from the CHMP is the final step before formal approval to market Naglazyme in the 25 member states of the European Union, Iceland and Norway. The CHMP has advised BioMarin that the committee's opinion will be forwarded to the European Commission (EC), which is expected to make a formal decision on marketing authorization within three to four months. The EC generally follows the advice of the CHMP, but it is not obligated to do so.

The CHMP is a scientific body made up of representatives from the 25 member states of the European Union, Iceland and Norway. The CHMP reviews medicinal product applications on their scientific and clinical merit, and provides advice on their approval to the EC, including recommendations about product labeling.

The CHMP reviewed BioMarin's clinical data for Naglazyme, which the company submitted in December 2004. The data included were from a 24-week, Phase 3, multi-center, double-blind, placebo-controlled trial involving 39 patients, of which one patient from the placebo group dropped out of the trial for reasons unrelated to treatment, and 24 weeks of data from the extension study, which involved all remaining 38 patients.

About MPS VI

MPS VI (also known as Maroteaux-Lamy syndrome) is a debilitating, life-threatening genetic disease caused by a deficiency of the enzyme N-acetylgalactosamine 4-sulfatase. This enzyme deficiency leads to the accumulation of certain complex carbohydrates, glycosaminoglycans (GAGs), in the lysosomes, giving rise to progressive cellular, tissue and organ system dysfunction. The majority of individuals with MPS VI die from disease-related complications between childhood and early adulthood. Additional information can be found at www.mpsvi.com.

About Naglazyme

Naglazyme is an enzyme replacement therapy for the treatment of MPS VI. On May 31, 2005, the U.S. Food and Drug Administration (FDA) granted market authorization of Naglazyme for patients with MPS VI. As the first drug approved for this indication, the FDA granted Naglazyme orphan drug status, which confers upon it seven years of market exclusivity. In Europe, regulatory authorities have designated Naglazyme as an orphan medicinal product, which, if it is the first therapy approved for MPS VI, will guarantee 10 years of market exclusivity within the European Union. Additional information can be found at www.naglazyme.com.

Naglazyme is indicated for patients with MPS VI. Naglazyme has been shown to improve walking and stair-climbing capacity.

The most common adverse events observed in clinical trials in Naglazyme- treated patients were headache, fever, arthralgia, vomiting, upper respiratory infections, abdominal pain, diarrhea, ear pain, cough, and otitis media. Severe reactions included angioneurotic edema, hypotension, dyspnea, bronchospasm, respiratory distress, apnea, and urticaria. The most common symptoms of infusion reactions included fever, chills/rigors, headache, rash, and mild to moderate urticaria. Nausea, vomiting, elevated blood pressure, retrosternal pain, abdominal pain, malaise, and joint pain were also reported. No patients discontinued Naglazyme infusions for adverse events and all patients that completed the double-blind portion of the trial continue to receive weekly infusions of Naglazyme. Nearly all patients developed antibodies as a result of treatment, but the level of the immune response did not correlate with the severity of adverse events or impact the improvements experienced in endurance. Because antihistamine use may increase the risk of apneic episodes, evaluation of airway patency should be considered prior to the initiation of treatment. Consideration to delay Naglazyme infusion should be given when treating patients who present with an acute febrile or respiratory illness.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio is comprised of three approved products and multiple clinical and preclinical product candidates. Approved products include Naglazyme(TM) (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin, Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), and Orapred(R) (prednisolone sodium phosphate oral solution) for inflammatory conditions. Investigational product candidates include Phenoptin(TM) (sapropterin hydrochloride), a Phase 3 product candidate for the treatment of phenylketonuria (PKU). For additional information, please visit www.BMRN.com. 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 and commercialization of Naglazyme and Phenoptin; expectations related to post-marketing commitments for Naglazyme; and actions by regulatory authorities. 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: possible delays in launching Naglazyme in the E.U. and slow market penetration in the U.S. and E.U.; the content and timing of decisions by the FDA and European Commission and other regulatory authorities concerning Naglazyme and Phenoptin; issues or complications associated with post-marketing commitments; the results of current and future clinical trials of Phenoptin; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Factors That May Affect Future Results" in BioMarin's 2004 Annual Report on Form 10-K and the factors contained in BioMarin's reports on Forms 10-Q and 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.

NOTE: Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC.

Orapred(R) is a registered trademark of Medicis Pediatrics, Inc. and is used under license.


   Investors                                    Media
   Joshua A. Grass                              Susan Ferris
   Director, Business Development               Manager, Corporate
   & Finance                                    Communications
   BioMarin Pharmaceutical Inc.                 BioMarin Pharmaceutical Inc.
   415-506-6777                                 415-506-6701

SOURCE: BioMarin Pharmaceutical Inc.

CONTACT: investors, Joshua A. Grass, Director, Business Development &
Finance, +1-415-506-6777, or media, Susan Ferris, Manager, Corporate
Communications, +1-415-506-6701, both of BioMarin Pharmaceutical Inc.