BioMarin Provides Highlights Ahead of Research and Development Day

BioMarin Provides Highlights Ahead of Research and Development Day

  *Vimizim: Regulatory applications on track. FDA Advisory Committee Meeting
    expected to be scheduled for November 19, 2013.
  *BMN 701: Maximal Inspiratory Pressure (MIP) accepted by FDA as primary
    endpoint for Phase 3 Study in Late Onset Pompe Disease.
  *BMN 111: US and European Regulatory Authorities agree that Phase 2 Study
    in Achondroplasia can start without additional data.
  *BMN 190: BioMarin enrolls first patient in Phase 1/2 Study for the
    treatment of Neuronal Ceroid Lipofuscinosis Type 2, a form of Batten

SAN RAFAEL, Calif., Sept. 17, 2013 (GLOBE NEWSWIRE) -- BioMarin
Pharmaceutical Inc. (Nasdaq:BMRN) today announced highlights to be presented
at the company's Research and Development Day for the investment community to
be held today from 8:00 a.m. to 12:00 p.m. ET.Members of BioMarin's
management team and industry experts will provide an update on the company's
product portfolio and advancements in the research and development
pipeline.Interested parties may access a live audio webcast and slides of the
presentation via the investor section of the BioMarin website,

Vimizim for the Treatment of Morquio A

BioMarin announced today that it expects that Vimizim will be reviewed by the
U.S. Food and Drug Administration Advisory Committee on November 19, 2013. The
Company noted that the FDA has previously granted Vimizim priority review
designation and assigned a PDUFA date of February 28, 2014.The European
Medicines Agency (EMA) has validated the Vimizim Marketing Authorization
Application (MAA) and has granted accelerated review status, which could lead
to a (Committee for Medicinal Products for Human Use) CHMP opinion before year
end. During the second quarter of 2013, the company also submitted the
Marketing Authorisation (MA) in Brazil. Ongoing production of bulk drug
substance and drug product will allow for a global launch of Vimizim following
approvals in multiple countries. Both the FDA and EMA have conducted or
scheduled pre-approval inspections associated with the review of the marketing

BMN 701 for the Treatment of Pompe Disease

The company announced that it has received agreement from the FDA that Maximal
Inspiratory Pressure (MIP) may be utilized as the primary endpoint for its
anticipated Phase 3 switching trial with BMN-701, a fusion protein of
insulin-like growth factor 2 and acid alphaglucosidase (IGF2-GAA) for the
treatment of late-onset Pompe disease. Discussions with European regulators
regarding primary endpoint and study design are still ongoing.

Subject to completing discussions with European health authorities, the
company expects to initiate a Phase 3 switching trial by the fourth quarter of
2013 or the first quarter of 2014 in late-onset Pompe patients who have
previously been treated with alglucosidase alfa. The proposed study design is
a single-arm trial, with treatment at 20 mg/kg administered every other week
for 24 weeks. The company intends to use efficacy as measured by the
respiratory parameter MIP as the primary endpoint. MIP is the maximal pressure
that can be produced by the patient trying to inhale through a blocked
mouthpiece.Secondary objectives include MEP and six-minute walk test, as well
as safety. The study will be conducted with full scale material from a revised
manufacturing process, which has improved process robustness and increased

BMN 111 for the Treatment of Achondroplasia

The company announced today that based on recent meetings with the FDA and the
Medicines and Healthcare Products Regulatory Agency (MHRA) it will be able to
proceed directly to a Phase 2 study of BMN 111, an analog of C-type
Natriuretic Peptide (CNP), for achondroplasia. BioMarin previously completed a
Phase 1 study in adult healthy volunteers. The FDA had placed a partial
clinical hold on BMN 111, but at a recent meeting indicated that following
submission of a modified Phase 2 protocol, and withdrawal of the previous
Phase 2 study protocol, the clinical hold can be lifted and that no further
data is required before initiating a Phase 2 study. The company expects to
initiate its first global study in pediatric patients with achondroplasia in
the fourth quarter of 2013 or the first quarter of 2014.

BMN 190 for the Treatment of Batten Disease

The company announced today that it has initiated the Phase 1/2 study 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 first patient has been enrolled and is expected to receive
initial drug dosing within 7 days.

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 after 48 weeks of
treatment.Secondary objectives are to evaluate the impact of treatment on
brain atrophy in comparison with NCL-2 natural history after 48 weeks of
treatment and to characterize pharmacokinetics and immunogenicity.The study
will enroll approximately 22 subjects at up to ten clinical sites worldwide
for a treatment duration of 48 weeks.

BMN 673 for the Treatment of gBRCA Breast Cancer

With BMN 673 PARP inhibition with BMN 673 in ovarian and breast cancer
patients with deleterious mutations of BRCA1 and BRCA2 has been selected as a
late breaking abstract by the 17th ECCO – 38th ESMO – 32nd ESTRO European
Cancer Congress in Amsterdam, The Netherlands and will be presented in an oral
presentation on September 29, 2013.

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 III 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 III 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 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 our expectations regarding the design, progress and timing of
BioMarin's clinical trials of BMN-673, BMN-701, BMN-111 and BMN-190; our
expectations regarding the timing of the FDA Advisory Committee Meeting and
CHMP opinion for Vimizim; our expectations regarding the timing of our
regulatory filings for Vimizim, BMN-673, BMN-701, BMN-111 and BMN-190; actions
by regulatory authorities, particularly actions related to the approval of
Vimizim. 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: our success
in the continued commercialization of Naglazyme, Kuvan, and Firdapse; Genzyme
Corporation's success in continuing the commercialization of Aldurazyme;
results and timing of current and planned preclinical studies and clinical
trials, particularly with respect to BMN-673, BMN-701, BMN 190 and BMN-111;
our ability to successfully manufacture our products and product candidates
and to pass pre-approval inspections with the FDA and EMA related to the
approval of Vimizim; the content and timing of decisions by the U.S. Food and
Drug Administration, the European Commission and other regulatory authorities
concerning Vimizim and each of the other described products and product
candidates; and those risks that are discussedin BioMarin's filings with the
Securities and Exchange Commission, including, without limitation, BioMarin's
2012 Annual Report on Form 10-K, and our periodic reports on Form 10-Q and
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® and Firdapse™ are registered trademarks of
BioMarin Pharmaceutical Inc.

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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