BioMarin Acquires Zacharon Pharmaceuticals

BioMarin Acquires Zacharon Pharmaceuticals

            Lead Program Focused on the Oral Treatment of MPS III

    Acquisition Augments Existing Analytical Capabilities and Expertise in

SAN RAFAEL, Calif., Jan. 7, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) announced today that it has acquired Zacharon
Pharmaceuticals, a private biotechnology company based in San Diego focused on
developing small molecules targeting pathways of glycan and glycolipid

Zacharon drug discovery projects include two ongoing lead optimization
programs, inhibition of heparan sulfate synthesis for MPS III and other MPS
disorders, and inhibition of ganglioside synthesis for diseases such as Tay
Sachs and Sandhoff. Zacharon's proprietary SensiPro^® platform is a powerful
technology for analysis of specific carbohydrate structures and therefore the
identification of candidate drugs to treat those conditions.

Hank Fuchs, M.D., Executive Vice President and Chief Medical Officer of
BioMarin said, "Zacharon's lead program, focused on reducing the accumulation
of heparan sulfate, offers the exciting prospect of treating both the CNS and
peripheral manifestations of MPS III, and potentially other MPS disorders,
with an orally bioavailable small molecule. In general, reducing the synthesis
of the target substrate alleviates the burden on the compromised lysosomal
system, and this therapeutic approach has been clinically validated with other
enzyme inhibitors. Zacharon's deep expertise in glycobiology has generated
additional programs for treating lysosomal storage disorders that we expect to
progress, and we will leverage that expertise to continue to build BioMarin's
existing research and development pipeline into a sustainably leading

"The acquisition of Zacharon will further expand our glycobiology expertise
and will support our lysosomal storage disease drug development efforts," said
Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "We are committed
to investing in our advancing pipeline, which has evolved through a
combination of internal development and targeted acquisitions, such as this."

Under the terms of the stock purchase agreement, BioMarin paid $10 million
upfront for 100 percent of Zacharon's share capital and may make potential
additional payments for clinical, regulatory and commercial milestones. The
costs to be incurred in 2013 of acquiring Zacharon and absorbing its
operations is covered by the previously provided 2013 R&D expense guidance.

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 completedPhase 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.

The BioMarin Pharmaceutical Inc. logo is available at

Forward-Looking Statement

This press release contains forward-looking statements about the business
prospects of BioMarin Pharmaceutical Inc., including, without limitation,
statements about: expectations related to the Zacharon development programs,
and the development of BioMarin's other programs, including GALNS; and actions
by regulatory authorities. 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; the content and timing of decisions by the U.S. Food and Drug
Administration, the European Commission and other regulatory authorities
concerning each of the described products and 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 2011 Annual Report on Form 10-K, and the
factors contained in BioMarin's reports on Form 10-Q. 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
         Debra Charlesworth
         BioMarin Pharmaceutical Inc.
         (415) 455-7451

BioMarin Pharmaceutical Inc. Logo
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