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BioMarin Submits CTA for BMN-190 for Batten Disease

BioMarin Submits CTA for BMN-190 for Batten Disease

SAN RAFAEL, Calif., April 1, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) announced today that it has submitted a Clinical Trial
Application (CTA) with the Medicines and Healthcare Products Regulatory Agency
(MHRA) in the U.K. for BMN-190, a recombinant human tripeptidyl peptidase 1
(rhTPP1) for the treatment of patients with neuronal ceroid lipofuscinosis
type 2 (NCL-2), a form of Batten disease. The company expects to start
enrolling patients in a Phase 1/2 trial in mid-2013.

"The Batten disease program embodies our core mission at BioMarin -- to
develop life-altering therapies for ultra-orphan diseases with a significant
and serious unmet medical need," said Hank Fuchs, M.D., Executive Vice
President and Chief Medical Officer of BioMarin. "We hope to leverage our
expertise in enzyme replacement therapy development to advance this program
and bring a therapeutic option to patients suffering from NCL-2. We have
demonstrated encouraging pharmacological activity in preclinical models and
hope to see this benefit translated into human subjects."

The Phase 1/2 study is an open-label, dose-escalation study in patients with
NCL-2. The primary objectives are to evaluate the safety and tolerability of
BMN-190 and to evaluate effectiveness using an NCL-2-specific rating scale
score in comparison with natural history data.Secondary objectives are to
evaluate the impact of treatment on brain atrophy in comparison with NCL-2
natural history, and to characterize the pharmacokinetics and immunogenicity.
The study will enroll approximately 22 subjects worldwide for a treatment
duration of 48 weeks.

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
(N-acetylgalactosamine 6-sulfatase), 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, BMN-111, a modified
C-natriuretic peptide, which is currently in Phase I clinical development for
the treatment of achondroplasia, and BMN-190, a recombinant human tripeptidyl
peptidase 1 (rhTPP1), which is currently in Phase 1/2 development for the
treatment of neuronal ceroid lipofuscinosis Type 2 disease. 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: the development of BioMarin's BMN-190 program generally and
the timing and results of the planned Phase 1/2 trial of BMN-190. 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 preclinical studies and clinical trials of BMN-190; the content
and timing of decisions by the U.S. Food and Drug Administration, the European
Commission and other regulatory authorities; 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 Annual Report on Form 10-K for the Year ended December 31, 2012.
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® and 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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