Vimizim Marketing Application Submitted to ANVISA in Brazil

Vimizim Marketing Application Submitted to ANVISA in Brazil

SAN RAFAEL, Calif., July 1, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) today announced that it has submitted the Vimizim Marketing
Application (MA) to the Agência Nacional de Vigilancia Sanitaria (ANVISA), or
the National Health Surveillance Agency Brazil. Assuming priority review
status, based on orphan drug status in the U.S. and EU, an approval decision
from ANVISA is anticipated in mid 2014.

"Considering that Brazil, along with the U.S., are expected to be the largest
market opportunities for Vimizim, the timely submission in this market is an
important milestone in our efforts to bring the first therapeutic option to
patients with Morquio A Syndrome," said Jean-Jacques Bienaimé, Chief Executive
Officer of BioMarin. "Along with orphan drug designation in the U.S. and EU,
priority review status in the U.S. and accelerated assessment status in the
EU, we are making good progress on a global basis. In the coming months, we
will continue to work closely with regulatory authorities and prepare for the
launch of Vimizim worldwide."

About MPS IVA

Mucopolysaccharidosis IVA (MPS IVA, also known as Morquio A Syndrome) is a
disease characterized by deficient activity of
N-acetylgalactosamine-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.

The rate of incidence of MPS IVA is as yet unconfirmed and varies among
different populations but estimates vary between 1 in 200,000 live births and
1 in 250,000 live births. The estimated prevalence is approximately 3,000
patients in the developed world.Based on knowledge of the worldwide
distribution of the MPS VI market and the more than 1,300 identified MPS IVA
patients worldwide, the company estimates that approximately 20 percent of
patients are in North America (15 percent in the U.S.) and approximately 50
percent of patients are in EUMEA.

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 BMN-110 (elosulfase alfa), formally referred to as GALNS,
which successfully completed Phase III clinical development for the treatment
of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which
is currently in Phase III 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, 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, and BMN-111, a modified
C-natriuretic peptide, which is currently in Phase I clinical development for
the treatment of achondroplasia. 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: expectations regarding the MA filing for Vimizim with
ANVISA; the potential outcome of the review of such filing; and the possible
approval of such product candidate.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 clinical
trials of its product candidates; the nature of the ANVISA's questions
associated with the MA and BioMarin's ability to timely respond to those
questions; BioMarin's ability to continue to meet the requirements for
accelerated assessment; ANVISA's compliance with it internal review
guidelines; the content and timing of decisions by ANVISA; 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, 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.

Vimizim™ is our trademark, and BioMarin®, Naglazyme®, Kuvan®, Firdapse® are
registered trademarks of BioMarin Pharmaceutical Inc.

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

CONTACT: Investors:
         Eugenia Shen
         BioMarin Pharmaceutical Inc.
         (415) 506-6570
        
         Media:
         Debra Charlesworth
         BioMarin Pharmaceutical Inc.
         (415) 455-7451

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