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Press release from GlobeNewswire (a Nasdaq OMX company)

FDA Posts Briefing Documents for Advisory Committee Meeting Reviewing Vimizim(TM) for the Treatment of Morquio A Syndrome

Friday, November 15, 2013

FDA Posts Briefing Documents for Advisory Committee Meeting Reviewing Vimizim(TM) for the Treatment of Morquio A Syndrome

06:15 EST Friday, November 15, 2013

SAN RAFAEL, Calif., Nov. 15, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the U.S. Food and Drug Administration (FDA) posted briefing materials for the November 19, 2013 Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) meeting to review the Vimizim™ (elosulfase alfa) Biologics License Application (BLA). Vimizim, an enzyme replacement therapy, is being developed for the treatment of patients with the lysosomal storage disorder Morquio A syndrome, also called Mucopolysaccharidosis Type IVA (MPS IVA).

The EMDAC meeting is scheduled for November 19, 2013 at 8:00am 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

The Prescription Drug User Fee Act date for completion of the review of the company's BLA for Vimizim is February 28, 2014.

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® (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 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. 

CONTACT: Investors:
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media:
Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451

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