BioMarin Doses First Patient in Phase 2 Trial With BMN 111 for the Treatment of Children With Achondroplasia

BioMarin Doses First Patient in Phase 2 Trial With BMN 111 for the Treatment
of Children With Achondroplasia

SAN RAFAEL, Calif., Jan. 14, 2014 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) announced today that it has dosed the first child in the
Phase 2 trial with BMN 111, an analog of C-type Natriuretic Peptide (CNP), for
the treatment of children with achondroplasia. Achondroplasia is the most
common form of disproportionate short stature or dwarfism.

"BMN 111 is representative of BioMarin's core competency of developing
life-altering therapies that address unmet medical needs," stated Hank Fuchs,
M.D., Chief Medical Officer of BioMarin."In this Phase 2 study, we hope to
see improvements in bone growth similar to what was observed in our
preclinical models, and resulting improvements in the medical complications of
achondroplasia that occur as a result of disproportionate bone growth.We
believe treatment with BMN 111 for achondroplastic children will be
well-tolerated and could potentially address the underlying cause of this
condition and lead to benefits in the lives of these patients."

The Phase 2 study is an open-label, sequential cohort, dose-escalation study
of BMN 111 in children who are 5-14 years old.The primary objective of this
study is to assess the safety and tolerability of daily subcutaneous doses of
BMN 111 administered for 6 months.The secondary objectives will include an
evaluation of change in annualized growth velocity, changes in absolute growth
parameters, changes in body proportions and other medically relevant and
functional aspects of achondroplasia, such as sleep apnea and joint range of
motion.Prior to enrolling in the Phase 2 study, all patients will have
participated in a 6 month natural history study to determine baseline growth
velocity data.This is an international study that will enroll approximately
24 subjects for a treatment duration of 6 months.

About Achondroplasia

Achondroplasia is the most common form of human dwarfism and is characterized
by failure of normal conversion of cartilage into bone. It is caused by an
autosomal dominant activating mutation in the fibroblast growth factor
receptor 3 (FGFR3) gene, a negative regulator of bone growth. Eighty percent
of cases are the result of a spontaneous mutation, and ninety-eight percent of
those cases have a G380R mutation. Clinical manifestations of the disease
include short stature, cervico-medullary compression, sleep apnea, bowed legs,
frontal bossing and mid-face hypoplasia, permanent sway of the lower back,
spinal stenosis, recurrent ear infections and obesity, all of which are
related to the disproportionate growth which is characteristic of the

The rate of incidence of achondroplasia is one in 15,000 to one in 40,000 live
births, with approximately 18,000 to 24,000 people in the U.S. and Europe

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 MPS VI, a
product wholly developed and commercialized by BioMarin; Aldurazyme®
(laronidase) for 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) with 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 development of BioMarin's BMN 111 program generally and
the timing and results of the planned Phase 2 trial of BMN 111. 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 111; the content
and timing of decisions by the U.S. Food and Drug Administration, the European
Commission and other regulatory authorities; our ability to successfully
manufacture the product candidate for the preclinical and clinical trials; 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.

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

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