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All Eyes On BioMarin Pharmaceutical Inc. (BMRN) As FDA Reviews Rare Disease Drug, Stock Trading Halted Today


11/19/2013 7:52:23 AM

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BioMarin Stock Trading Halted Today

SAN RAFAEL, Calif., Nov. 19, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (BMRN) announced today that NASDAQ has halted trading of the company's stock. The U.S. Food and Drug Administration's (FDA) Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) is meeting today to review BioMarin's Biologics License Application (BLA) for Vimizim, an enzyme replacement therapy being developed for the treatment of patients with the lysosomal storage disorder Morquio A syndrome. Morquio A syndrome, also called Mucopolysaccharidosis Type IVA (MPS IVA), is a rare, severely debilitating disease that substantially limits both the quality and length of life of those affected. There is currently no approved treatment for Morquio A syndrome, representing a significant unmet need for patients and their families.

The Advisory Committee meeting is scheduled for 8:00 a.m. ET. The briefing materials and webcast information can be found on the FDA website at:

http://www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/EndocrinologicandMetabolicDrugsAdvisoryCommittee/ucm331504.htm

About Morquio A Syndrome

Mucopolysaccharidosis IVA (MPS IVA, also known as Morquio A syndrome) is a disease characterized by deficient activity of Nacetylgalactosamine- 6-sulfatase (GALNS) causing excessive lysosomal storage of glycosaminoglycans such as keratan sulfate and chondroitin sulfate. This excessive storage causes a systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance. Malformation of the chest impairs respiratory function, and looseness of joints in the neck cause spinal instability and potentially spinal cord compression. Other symptoms may include hearing loss, corneal clouding, and heart disease. Initial symptoms often become evident in the first five years of life. The disease substantially limits both the quality and length of life of those affected.

About BioMarin

BioMarin 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(R) (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse(R) (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include Vimizim (N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which successfully completed Phase 3 clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase 3 clinical development for the treatment of PKU, BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase 3 clinical development for the treatment of germline BRCA breast cancer, BMN 701, a novel fusion of acid alpha glucosidase (GAA) with a peptide derived from insulin like growth factor 2, which is currently in Phase 1/2 clinical development for the treatment of Pompe disease, BMN 111, a modified C-natriuretic peptide, which is currently in Phase 1 clinical development for the treatment of achondroplasia and BMN 190, a recombinant human tripeptidyl peptidase-1 (rhTPP1) for the treatment of late-infantile neuronal ceroid lipofuscinosis (CLN2), a form of Batten Disease. 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 advisory committee meeting timing and the timing of the trading of BioMarin's common stock. 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 conduct and completion of the advisory committee meeting; action by NASDAQ; 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 2012 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(R), Naglazyme(R), Kuvan(R) and Firdapse(TM) are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC.

Contact:

Investors:

Traci McCarty

BioMarin Pharmaceutical Inc.

(415) 455-7558

Media:

Debra Charlesworth

BioMarin Pharmaceutical Inc.

(415) 455-7451

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