Naglazyme Approved by Japanese Ministry of Health

NOVATO, Calif., March 31 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical
Inc. (Nasdaq and SWX: BMRN) announced today that AnGes MG, Inc. (AnGes),
BioMarin's marketing and distribution partner in Japan, has received approval
for its Marketing Application for Naglazyme(R) (galsulfase) from the Japanese
Ministry of Health, Labour and Welfare (MHLW) for the treatment of patients
with Mucopolysaccharidosis VI ( MPS VI).
"We are proud to work with AnGes in bringing the first drug treatment
option to MPS VI patients in Japan," said Stephen Aselage, Senior Vice
President of Global Commercial Development at BioMarin. "We are dedicated to
providing life-altering therapies to patients around the world and continue to
expand our geographic footprint through our patient identification and
commercialization efforts."
BioMarin established a marketing and distribution agreement with AnGes in
December 2006, through which AnGes obtained exclusive rights to market
Naglazyme in the Japanese market. AnGes submitted a marketing application to
the MHLW in August 2007. Naglazyme was approved by the U.S. Food and Drug
Administration (FDA) in May 2005 and by the European Commission (EC) in
January 2006. As the first drug approved for MPS VI, the FDA and EC have both
designated Naglazyme as an orphan drug, conferring seven years of market
exclusivity in the United States and 10 years of market exclusivity in the
European Union. In addition, Naglazyme obtained orphan designation in June
2007 from the MHLW in Japan.
About MPS VI
MPS VI (also known as Maroteaux-Lamy syndrome) is a debilitating, life-
threatening genetic disease caused by a deficiency of the enzyme N-
acetylgalactosamine 4-sulfatase. This enzyme deficiency leads to the
accumulation of certain complex carbohydrates, glycosaminoglycans (GAGs), in
the lysosomes, giving rise to progressive cellular, tissue and organ system
dysfunction. The majority of individuals with MPS VI die from disease-related
complications between childhood and early adulthood. Additional information
can be found at http://www.mpsvi.com.
About Naglazyme
Naglazyme is the first and only enzyme replacement therapy indicated for
the treatment of MPS VI. Naglazyme is indicated for patients with MPS VI.
Naglazyme has been shown to improve walking and stair-climbing capacity.
The most common adverse events observed in clinical trials in Naglazyme-
treated patients were headache, fever, arthralgia, vomiting, upper respiratory
infections, abdominal pain, diarrhea, ear pain, cough, and otitis media.
Severe reactions included angioneurotic edema, hypotension, dyspnea,
bronchospasm, respiratory distress, apnea, and urticaria. The most common
symptoms of infusion reactions included fever, chills/rigors, headache, rash,
and mild to moderate urticaria. Nausea, vomiting, elevated blood pressure,
retrosternal pain, abdominal pain, malaise, and joint pain were also reported.
No patients discontinued for adverse events and all patients who completed the
double-blind portion of the trial continued to receive weekly infusions of
Naglazyme. Nearly all patients developed antibodies as a result of treatment,
but the level of the immune response did not correlate with the severity of
adverse events. Because antihistamine use may increase the risk of apneic
episodes, evaluation of airway patency should be considered prior to the
initiation of treatment. Consideration to delay Naglazyme infusion should be
given when treating patients who present with an acute febrile or respiratory
illness. Additional information can be found at http://www.naglazyme.com.
About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for
serious diseases and medical conditions. The company's product portfolio
comprises three approved products and multiple clinical and preclinical
product candidates. Approved products include Naglazyme(R) (galsulfase) for
mucopolysaccharidosis VI (MPS VI), a product wholly developed and
commercialized by BioMarin; Aldurazyme(R) (laronidase) for
mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a
50/50 joint venture with Genzyme Corporation; and Kuvan(TM) (sapropterin
dihydrochloride) Tablets, a product for the treatment of phenylketonuria
(PKU), developed in partnership with Merck Serono, a division of Merck KGaA of
Darmstadt, Germany. Other product candidates include 6R-BH4 for cardiovascular
indications, which is currently in Phase 2 clinical development for the
treatment of peripheral arterial disease and sickle cell disease, and PEG-PAL
(PEGylated recombinant phenylalanine ammonia lyase) for the treatment of PKU.
For additional information, please visit http://www.BMRN.com. Information on
BioMarin's website is not incorporated by reference into this press release.
About AnGes MG, Inc.
AnGes MG, Inc., a biopharmaceutical company founded in 1999, was
established on an innovative discovery by researchers of Osaka University. The
company specializes in research, development and practical application of
genetic medicine. Current programs include the Hepatocyte Growth Factor (HGF)
genetic medicine which improves blood circulation by regenerating blood
vessels, and an NFkB decoy that controls various types of inflammation.
Forward-Looking Statement
This press release contains forward-looking statements about the business
prospects of BioMarin Pharmaceutical Inc., including, without limitation,
statements about: the continued clinical development and commercialization of
Naglazyme and BioMarin's other products and product candidates 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:
our success in the continued commercialization of Naglazyme; 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 2006 Annual Report on Form 10-K, as amended, and the factors
contained in BioMarin's 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(R) and Naglazyme(R) are a registered trademarks of BioMarin
Pharmaceutical Inc.
Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC.

Contact:

Investors Media
Eugenia Shen Susan Berg
BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc.
(415) 506-6570 (415) 506-6594

SOURCE BioMarin Pharmaceutical Inc.

Investors, Eugenia Shen, +1-415-506-6570, or Media, Susan Berg,
+1-415-506-6594, both of BioMarin Pharmaceutical Inc.