Vimizim MAA Validated by the EMA

Vimizim MAA Validated by the EMA

Vimizim Designated for Accelerated Assessment by the EMA

SAN RAFAEL, Calif., May 30, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) today announced that the European Medicines Agency (EMA)
has validated the Marketing Authorization Application (MAA) for Vimizim.
Validation of the MAA confirms that the submission is complete and starts the
EMA's formal review process. Earlier this year, the EMA accepted BioMarin's
request for accelerated assessment for this MAA on the grounds that Vimizim
could satisfy an unmet medical need and is of major interest from the point of
view of therapeutic innovation and public health.Accelerated assessment has
the potential to shorten EMA's review procedure.However, at any time during
the MAA assessment, the EMA may decide to continue the assessment under
standard assessment timelines. Assuming the Vimizim application remains on the
accelerated assessment timeline, a CHMP opinion is anticipated in December
2013, and, if positive, a decision from the European Commission could be
received in the first quarter of 2014.

"With approximately half of all MPS IVA patients living in Europe, the Middle
East and Africa, this 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 the
acceptance of the BLA by the FDA, we are pleased to enter the review phase on
a global basis.MPS IVA represents a significant unmet medical need for those
affected, and our hope is to offer a life-altering treatment option to
patients 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 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, 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 theMAA for Vimizim with the EMA; 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 EMA's questions associated with the MAA and
BioMarin's ability to timely respond to those questions; BioMarin's ability to
continue to meet the requirements for accelerated assessment; the EMA's
compliance with it internal review guidelines; the content and timing of
decisions by the EMA; 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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