BioMarin Submits Vimizim BLA to the U.S. FDA for the Treatment of MPS IVA

BioMarin Submits Vimizim BLA to the U.S. FDA for the Treatment of MPS IVA

SAN RAFAEL, Calif., April 1, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) announced today the submission of a Biologics License
Application (BLA) to the U.S. Food and Drug Administration (FDA) for Vimizim
(BMN-110, elosulfase alfa), an enzyme replacement therapy under evaluation for
the treatment of patients with the rare lysosomal storage disorder
Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome. The
company intends to submit an application for registration in the European
Union (EU) by the end of April 2013.

"Based on the positive results from our Phase 3 pivotal study, we believe that
Vimizim offers a substantial benefit to patients with MPS IVA, a severely
debilitating and progressive disease for which there is no current treatment,"
said Hank Fuchs, M.D., Chief Medical Officer of BioMarin."The submission of
the BLA represents a significant milestone for BioMarin and is the result of
the strong, collaborative effort of many hard working employees,
investigators, patients, and their families.With this application, BioMarin
continues in its long-standing tradition of developing important therapies for
those who are most in need. We look forward to working with the U.S.
regulatory authorities to bring this treatment to patients."


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 between 1,000 and 1,500
patients in the U.S., EU and Japan and between 1,500 to 2,000 patients in the
rest of the world for a total of 2,500 to 3,000 patients.

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 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 BLA filing for Vimizim with the
FDA and the EMA; the potential outcome of the review of such filings; and the
possible approval of such product candidates.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 content and timing of decisions by the
U.S. Food and Drug Administration, the European Medicines Agency and other
regulatory authorities concerning its product candidates; 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.

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

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

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

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