2007 Annual Report
Accelerating Development. Delivering Breakthrough Treatments Worldwide.
BIOMARIN PHARMACEUTICAL INC.
page 1
Th ree products in 10 years. Since it was founded in 1997, BioMarin has
successfully advanced three breakthrough products from research through
FDA approval to patients – a remarkable accomplishment in the pharmaceutical
industry. By cultivating expertise in research, development and manufacturing,
and by streamlining clinical and regulatory development, BioMarin is providing
rapid access to treatment and support services to patients around the world
suff ering from rare inherited metabolic diseases.
Th is is Liam. He is one of hundreds of patients in the U.S. now enjoying the
benefi ts of Kuvan™, the fi rst FDA-approved treatment for phenylketonuria
(PKU). PKU patients cannot break down phenylalanine (Phe), an amino
acid found in food, which leads to high blood Phe levels that are toxic to the
brain. When left untreated, this can result in mental retardation and other
neurological problems. Before taking Kuvan, Liam struggled to keep his
blood Phe levels under control but now he’s better able to manage his PKU.
2007 Annual Report
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BIOMARIN PHARMACEUTICAL INC.
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Preclinical
Phase 1
Phase 2
Phase 3
BLA/NDA/MAA Commercialized
Pipeline
Naglazyme® (For MPS VI)
Aldurazyme® (For MPS I)
Kuvan™ (For PKU)
6R-BH4 (For CV Indications & Sickle Cell Disease)
PEG-PAL (For PKU)
BMN-174 (Small molecule correctors for Cystic Fibrosis)
BMN-175 (Small molecule potentiators for Cystic Fibrosis)
BMN-185 (IgA Protease for IgA Nephropathy)
Other preclinical products
Financial Highlights
Record Level Stock Performance
BioMarin’s stock continues to outperform the Nasdaq composite and the Nasdaq
biotech index. In December 2007, BMRN was bolstered by a 28.7 percent increase
following the FDA approval of Kuvan and closed the year with a 116 percent
increase overall.
Growing Market Capitalization
This increase in BioMarin’s valuation is a refl ection of the company’s
strength of its core business – three growing products serving high
unmet need populations, limited competitive threats and fi nancial
performance seen only among top tier biotech companies.
$3,394
$500
$400
$300
$200
$100
$1,386
$742
2002
2003
2004
2005
2006
2007
BioMarin
Nasdaq Composite Nasdaq Biotech Index
$411
2004
2005
2006
2007
I would like to thank our patients, their families and
physicians, and our corporate partners for helping
to bring these important life-altering therapies
to market. Thanks also to all of our employees
and our Board of Directors for their hard work,
dedication and passion for the patients we serve.
Together, they have brought three novel products
to market and continue to prove our expertise in
providing high-value biopharmaceuticals to the
patients who need them most. We appreciate your
continued support and look forward to keep-
ing you updated on our progress throughout the
coming year.
Sincerely,
Jean-Jacques Bienaimé
A Message from the CEO
Th e year 2007 marked many
signifi cant milestones for
BioMarin Pharmaceutical.
In addition to growing
product revenues and an
advancing pipeline, we
received FDA approval for
Kuvan, our third product
and the fi rst drug therapy
approved for the treatment
of phenylketonuria (PKU).
At remarkable speed, just over three years after
fi ling the IND, we are now delivering Kuvan to
hundreds of patients in the U.S., and together
with our partner Merck Serono, we look forward
to the approval and launch of the product in
Europe and abroad.
BioMarin is a fully-integrated multinational
commercial biopharmaceutical company with
the expertise, resources and dedication to
address serious unmet medical needs. We are
committed to providing life-altering therapies to
patients around the world.
Increasing global revenues In 2007, sales
of both Naglazyme and Aldurazyme grew at
impressive rates, driven by the identifi cation
of new patients and the active efforts of our
global commercial operations team to expand
geographic reach. Aldurazyme sales increased
28.5 percent over sales in 2006, and BioMarin’s
profi t in BioMarin/Genzyme LLC increased
58.0 percent over 2006. Looking forward, the
restructuring of the joint venture at the begin-
ning of 2008 will provide increased clarity to
our fi nancial reporting and result in more
effi cient operations.
Outstanding performance of Naglazyme, our
fi rst independently manufactured and com-
mercialized product, continues to demonstrate
that BioMarin has the expertise to successfully
develop and commercialize products, which
positions us as a strong stand-alone company.
Sales of Naglazyme grew 85.4 percent
compared to 2006 and we expect continued
growth as we begin to provide product to
additional regions of the world with high concen-
trations of MPS VI patients, such as Latin America
and Turkey.
We expect that Kuvan, along with Aldurazyme and
Naglazyme, will drive improvements in our bottom
line in 2008 and beyond. As a result, we are
expecting to reach full year profi tability in 2008, a
signifi cant milestone for BioMarin. Furthermore,
our record of proven commercial success,
coupled with our strong cash position, enables
us to pursue additional in-licensing or acquisition
opportunities to augment our clinical stage pipe-
line and ensure the continuation of double-digit
revenue growth in the coming years.
Advancing Product Pipeline Growing product
revenues from Naglazyme, Aldurazyme and now
Kuvan will help advance ongoing research and
development efforts to strengthen our product
pipeline. In 2007, we fi led the investigational new
drug (IND) application for PEG-PAL, (PEGylated
recombinant phenylalanine ammonia lyase), an
enzyme substitution therapy for the treatment of
PKU, and the Phase I trial is expected to begin in
the fi rst half of 2008. We are hopeful that the posi-
tive preclinical data showing sustained decreases
in blood Phe levels in PKU mice will be replicated
in humans. If proven safe and effective, PEG-PAL
has tremendous potential and may have the ability
to treat the entire spectrum of PKU patients by
bringing patients’ Phe levels down to normal levels.
In addition to its role in the treatment of PKU, 6R-
BH4 is an enzyme co-factor linked to maintaining
healthy endothelial function. To that end, we are
performing a number of Phase 2 and exploratory
studies to clarify its role in the treatment of various
cardiovascular indications and sickle cell disease.
Our pipeline also includes compounds in devel-
opment for the treatment of cystic fi brosis, IgA
nephropathy and other rare diseases. Our goal is
to fi le one IND per year.
Looking Ahead in 2008 We are proud of our
many achievements of 2007. The launch
of Kuvan represents an important
milestone for both BioMarin and the
PKU community and while we are
extremely pleased to have delivered
this promising treatment to market,
we also recognize the need to
continually deliver additional value.
To that end, we are investing in our
internal pipeline and aggressively
but prudently pursuing in-licensing
and acquisition opportunities. We
remain passionately dedicated to
serving MPS I, MPS VI and PKU
patients and we continue seek-
ing new ways to enhance value
to both patients and investors.
BIOMARIN PHARMACEUTICAL INC.
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2007 Annual Report
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BIOMARIN PHARMACEUTICAL INC.
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2007 Annual Report
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Delivering Breakthrough Treatments Worldwide
BioMarin is providing important therapeutic
treatments to hundreds of patients around the
world who might otherwise go untreated. In 2007,
the company continued to solidify its commercial
presence in the United States and Europe, and is
rapidly expanding into other regions such as Latin
America and the Middle East where the incidence
of MPS VI is more widespread.
As a fully-integrated multinational commercial
biotechnology company, BioMarin is leveraging its
expertise in manufacturing, research, regulatory
affairs and global commercial development, and
is making signifi cant strides in delivering fi rst-to-
market therapeutic treatments to patients with rare
genetic diseases.
In 2007 BioMarin received FDA
approval for its third product,
Kuvan (sapropterin dihydrochlo-
ride) Tablets, the fi rst drug therapy
approved for the treatment of PKU.
The company also fi led an IND and
is preparing to initiate clinical trials
for PEG-PAL (PEGylated recom-
binant phenylalanine ammonia lyase) to address
the entire spectrum of PKU patients who do not
respond to, or wish to reduce blood Phe levels
beyond what is possible with Kuvan.
Addressing new market opportunities. BioMarin
is working to further diversify its product portfolio
with various research and development programs.
For example, BH4, the active ingredient in Kuvan,
is currently being evaluated for a number of other
indications such as sickle cell disease, pulmo-
nary arterial hypertension and peripheral arterial
disease. A variety of other promising preclinical
programs are also being pursued with the goal
of fi ling one IND per year to ensure continued
top-line growth.
Building strength in numbers. During 2007,
BioMarin expanded its employee population by
25 percent in support of the global commercializa-
tion of Naglazyme®, the approval and launch of
Kuvan, and the expansion of multiple research
and development and clinical programs. Strong
employment growth is expected to continue in
the foreseeable future.
BioMarin’s active global commercialization efforts
are yielding hundreds of new MPS VI patients
who can benefi t from Naglazyme® (galsulfase),
the company’s fi rst independently developed and
commercialized enzyme replacement
therapy for mucopolysaccharidosis
VI. Worldwide sales of the product
increased more than 85 percent
in 2007 and are expected to grow
to more than $105 million in the
coming year.
In 2007, profi tability of the BioMarin/
Genzyme LLC joint venture of
Aldurazyme (laronidase for MPS I) grew
at a rapid rate. As a result of the restruc-
turing of the 50/50 joint venture, which
became effective at the beginning of
2008, each company is now better able
to maximize respective management and opera-
tional effi ciencies related to the product.
Accelerating commercial success. Kuvan was
approved in just a little over three years after the
IND fi ling and was launched within days of its
approval by the FDA. Similarly, both Naglazyme
and Aldurazyme were successfully developed
and commercialized in about fi ve years – a rate far
surpassing that of industry standards. BioMarin
is also aggressively pursuing ways to maximize
its intellectual property position and signifi cantly
extend product exclusivity beyond the orphan
protection timelines in the U.S. and abroad.
BioMarin patients. BioMarin is committed to
providing life-altering therapies to patients around
the world who suff er from rare genetic diseases.
2007 Annual Report
page 8
Corporate Information
BioMarin develops and commercializes innovative biopharmaceuticals
for serious diseases and medical conditions. Th e company’s portfolio
comprises three approved products and multiple clinical and preclinical
product candidates.
Executives
Jean-Jacques Bienaimé
Chief Executive Offi cer
Jeffrey H. Cooper
Senior Vice President,
Chief Financial Offi cer
Emil D. Kakkis, M.D., Ph.D.
Senior Vice President,
Chief Medical Offi cer
Steven Jungles
Vice President,
Supply Chain
Daniel P. Maher
Vice President,
Product Development
Charles A. O’Neill, Ph.D.
Vice President,
Pharmacological Sciences
Stephen Aselage
Senior Vice President,
Global Commercial Development
R. Andrew Ramelmeier, Ph.D.
Vice President,
Manufacturing and Process Development
Robert A. Baffi , Ph.D.
Senior Vice President,
Technical Operations
Stuart J. Swiedler, M.D., Ph.D.
Senior Vice President,
Clinical Affairs
G. Eric Davis
Vice President,
General Counsel & Secretary
Mark Wood
Vice President,
Human Resources
Jeff Ajer
Vice President,
Sales & Marketing Operations
Luisa Bigornia
Vice President,
Intellectual Property
Lewis Chapman
Vice President,
Global Marketing
Steve Glass
Vice President,
General Manager, European Operations
Victoria Sluzky, Ph.D.
Vice President,
Quality and Analytical Chemistry
Gordon Vehar, Ph.D.
Vice President,
Research
Amy Waterhouse
Vice President,
Regulatory & Government Affairs
Board of Directors
Jean-Jacques Bienaimé
Chief Executive Offi cer
Michael Grey
President & Chief Executive Offi cer,
SGX Pharmaceuticals, Inc.
Elaine Heron
Chairman & Chief Executive Offi cer,
Labcyte Inc.
Joseph Klein, III
Managing Director,
Gauss Capital Advisors, LLC.
Pierre Lapalme
Former President &
Chief Executive Offi cer,
North America Ethypharm, Inc.
V. Bryan Lawlis
President & Chief Executive Offi cer,
Itero Biopharmaceuticals, Inc.
Alan Lewis
President & Chief Executive Offi cer,
Novocell, Inc.
Randy Meier
Executive Vice President of Operations,
President of Global Eye Care &
Chief Financial Offi cer,
Advanced Medical Optics, Inc.
Corporate Headquarters
BioMarin Pharmaceutical Inc.
105 Digital Drive
Novato, CA 94949
Tel. 415.506.6700
Fax. 415.382.7889
Email. ir@bmrn.com
www.bmrn.com
BioMarin Europe Ltd.
Axtell House
23-24 Warwick Street
London W1B 5NQ
UK
BioMarin Brasil Farmacêutica
Rua James Joule, 92 – conjunto 42
Sao Paulo,
Brazil 04576-080
Stock Listing
BioMarin Pharmaceutical Inc. is listed
on the Nasdaq Global Market under the
symbol BMRN.
Independent Accountants
KPMG LLP
San Francisco, CA
Transfer Agent
Bank of New York Mellon
480 Washington Boulevard
Jersey City, NJ 07310
U.S. Tel. 800.522.6645
International Tel. 201.680.6578
Forward-Looking Statement: This Annual Report contains ‘forward-looking statements’ as defi ned under securities laws. These
statements can generally be identifi ed by the use of terminology such as ‘believes’, ‘expects’, ‘anticipates’, ‘plans’, ‘intends’, ‘may’,
‘will’, ‘projects’, ‘continues’, ‘estimates’, ‘potential’, ‘opportunity’, and so on. The company’s actual results or experience may differ
signifi cantly from the forward-looking statement. Factors that could cause or contribute to these differences include the results of
current clinical trials, the company’s ability to obtain regulatory approval for product candidates, its ability to successfully market
products and other factors discussed in the enclosed Form 10-K and the section entitled ‘Risk Factors’ therein.
One should not place undue infl uence on these forward-looking statements that speak only as of the date that they were made. These
cautionary statements should be considered in connection with any written or oral forward-looking statements that the company may
issue in the future. BioMarin Pharmaceutical Inc. does not undertake any obligation to release publicly any revisions to these forward-
looking statements after completion of the distribution of this Annual Report to refl ect later events or circumstances or to refl ect the
occurrence of unanticipated events.
Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC. Naglazyme® and BioMarin are registered trademarks of BioMarin
Pharmaceutical Inc. KUVAN™ is a trademark of BioMarin Pharmaceutical Inc.
BIOMARIN PHARMACEUTICAL INC.
105 Digital Drive, Novato, CA 94949
Tel: 415-506-6700 Fax: 415-382-7889
E-mail: ir@bmrn.com www.bmrn.com