BioMarin Initiates Phase 3 Trial for BMN 673 for the Treatment of Metastatic gBRCA Breast Cancer

BioMarin Initiates Phase 3 Trial for BMN 673 for the Treatment of Metastatic
gBRCA Breast Cancer

SAN RAFAEL, Calif., Oct. 31, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) announced today that it has dosed the first patient in its
Phase 3 program to evaluate BMN 673, its poly ADP-ribose polymerase (PARP)
inhibitor, in the treatment of metastatic germline BRCA mutated breast cancer.

"It has been very exciting to work with this novel molecule in the preclinical
laboratory where we saw it to be best in class. It is equally exciting to now
be involved in the clinical translation of this drug in this genetically
defined population of breast cancer patients who may benefit from a treatment
option specifically designed for them," said Dennis Slamon, Ph.D., M.D.,
Chief, Division Hematology/Oncology and Director of Clinical/Translational
Research, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine
at the University of California, Los Angeles. "Oncology therapy is moving
towards a personalized and targeted model with the goal of establishing more
effective treatments based on an individual's genetic profile, and BMN 673
could be an important step toward tailoring cancer treatments by tumor type."

"Enrolling the first patient is an important milestone in the clinical trial
process. We are excited to be part of a trial that not only offers the
potential to eliminate the use of chemotherapy, but also the possibility of
improving the length and quality of life for patients already diagnosed with
hereditary breast cancer," said Fran Visco, President of the National Breast
Cancer Coalition.

The Phase 3 study is an open-label, randomized, parallel, two-arm,
multi-center study of BMN 673 versus physician's choice in approximately 430
germline BRCA mutation patients with locally advanced and/or metastatic breast
cancer, who have received no more than two prior chemotherapy regimens for
metastatic disease.The primary objective of the study is to measure
progression free survival (PFS).Secondary objectives include evaluating the
objective response rate (ORR) and the overall survival (OS).

"We are eager to fully enroll this important trial for breast cancer patients
with hereditary breast cancer to better understand the role of BMN 673 in this
defined population," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin.
"We are looking forward to gaining a better understanding of the safety and
efficacy of our compound."

About Hereditary Breast Cancer with BRCA Mutation

BRCA1 and BRCA2 are human genes that belong to a class of genes known as tumor
suppressors. Mutation of these genes has been linked to hereditary breast and
ovarian cancer.A woman's risk of developing breast and/or ovarian cancer is
greatly increased if she inherits a deleterious (harmful) BRCA1 or BRCA2
mutation. Men with these mutations also have an increased risk of breast
cancer. Both men and women who have harmful BRCA1 or BRCA2 mutations may also
be at increased risk of other cancers.

Source:National Cancer Institute at the National Institutes of Health

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 Vimizim
(N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which
successfully completed Phase 3 clinical development for the treatment of MPS
IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is
currently in Phase 3 clinical development for the treatment of PKU, BMN 673, a
poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase 3
clinical development for the treatment of germline BRCA breast cancer, BMN
701, a novel fusion of acid alpha glucosidase (GAA) wth a peptide derived from
insulin like growth factor 2, which is currently in Phase 1/2 clinical
development for the treatment of Pompe disease, BMN 111, a modified
C-natriuretic peptide, which is currently in Phase 1 clinical development for
the treatment of achondroplasia and BMN 190, a recombinant human tripeptidyl
peptidase-1 (rhTPP1) for the treatment of late-infantile neuronal ceroid
lipofuscinosis (CLN2), a form of Batten Disease. 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: the enrollment of a Phase III trial for BMN 673 in
metastatic gBRCA breast cancer, the expectations of the development of BMN
673, including the possible safety and efficacy of such 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: the successful enrollment of
the Phase 3 trial; results and timing of current and planned preclinical
studies and clinical trials of BMN 673; our ability to successfully
manufacture BMN 673; the content and timing of decisions by the U.S. Food and
Drug Administration, the European Commission and other regulatory authorities
concerning BMN 673; 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, 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., or its affiliates.Aldurazyme^® is a registered
trademark of BioMarin/Genzyme LLC.

CONTACT: Investors
         Traci McCarty
         BioMarin Pharmaceutical Inc.
         (415) 455-7558
         Debra Charlesworth
         BioMarin Pharmaceutical Inc.
         (415) 455-7451

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