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BioMarin Initiates Phase 2 Study for GALNS in Patients Under Five Years of Age With MPS IVA

NOVATO, Calif., Nov. 29, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced the initiation of a Phase 2 study for GALNS (N-acetylgalactosamine 6-sulfatase) in patients under five years of age with mucopolysaccharidosis IVA (MPS IVA). 

"We expect this study to build our knowledge base in enzyme replacement therapies wherein clinicians believe earlier therapy is more impactful," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin.  "Comparatively speaking, this study is starting years before a similar study for Naglazyme, which was done as a post-marketing commitment.  We will also conduct a study in non-ambulatory patients, which, along with the study in patients under five years of age, will encompass the spectrum of MPS IVA patients not included in the Phase 3 trial.  Enrollment in the Phase 3 pivotal study is on track for results in the second half of 2012, and its execution is BioMarin's highest product development priority."

The primary objective of the Phase 2, open-label, multinational clinical study is to evaluate the safety and tolerability of infusions of GALNS at a dose of 2.0 mg/kg/week over a 52-week period in ten to fifteen MPS IVA patients under five years of age.  The secondary objectives are to evaluate urinary keratan sulfate (KS) levels and growth velocity.

About BioMarinBioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse™ (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase III clinical development for the treatment of MPS IVA, amifampridine phosphate (3,4-diaminopyridine phosphate), which is currently in Phase III clinical development for the treatment of LEMS in the U.S., PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU, BMN 701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, and BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers. For additional information, please visit Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking StatementThis press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the expectations related to the continued clinical development of its product candidate GALNS; 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:; results and timing of current and planned preclinical studies and clinical trials of GALNS; our ability to successfully manufacture our products and product candidates; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning GALNS 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 2010 Annual Report on Form 10-K, and the factors contained in BioMarin's reports on Form 10-Q. 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.

BioMarin®, Naglazyme®, Kuvan® and Firdapse™ are registered trademarks of BioMarin Pharmaceutical Inc.

Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.


 InvestorsMediaEugenia ShenBob PurcellBioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.(415) 506-6570

(415) 506-3267 




SOURCE BioMarin Pharmaceutical Inc.
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