FDA Accepts Vimizim BLA and Grants Priority Review Designation

FDA Accepts Vimizim BLA and Grants Priority Review Designation

PDUFA Action Date Extended to February 28, 2014

SAN RAFAEL, Calif., May 30, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) today announced that the U.S. Food and Drug Administration
(FDA) has accepted for review the Biologics License Application (BLA) 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

The FDA has granted priority review designation to Vimizim, which is granted
to drugs that offer major advances in treatment, or provide a treatment where
no adequate therapy exists.During the initial review of the application, the
FDA requested additional Chemistry, Manufacturing and Controls (CMC)
information.The company provided the information as requested, and the FDA
designated it as a major amendment to the application thus extending the PDUFA
action date by three months.The extended PDUFA action date is February 28,

In the FDA's filing communication, the Agency informed the company that it is
currently planning to hold an advisory committee meeting to discuss the
application.No date has been set for this meeting.

"We are pleased that the FDA has granted priority review to the Vimizim BLA,
and we look forward to a productive dialog with the FDA as they review the
application," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin.
"With our expertise in developing enzyme replacement therapies to treat
serious unmet medical needs, we plan to work closely with the FDA and other
regulatory authorities to bring this much needed therapy to MPS IVA 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 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 the regulatory process for the BLA
filing for Vimizim with the FDA; 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 FDA's
questions associated with the BLA and BioMarin's ability to timely respond to
those questions; the FDA's compliance with it internal review guidelines; the
outcome of the advisory committee meeting related to the BLA; the content and
timing of decisions by the U.S. Food and Drug Administration; 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
         Debra Charlesworth
         BioMarin Pharmaceutical Inc
         (415) 455-7451

BioMarin Pharmaceutical Inc. Logo
Press spacebar to pause and continue. Press esc to stop.