CORPORATE INFORMATION
EXPERIENCED MANAGEMENT
A MESSAGE FROM THE CEO
2006 Annual Report
Stock Performance
BioMarin develops and commercializes
innovative biopharmaceuticals for serious
diseases and medical conditions. The
company’s portfolio comprises two
approved products and multiple clinical
and preclinical product candidates.
Corporate Headquarters
Transfer Agent
BioMarin
Nasdaq Stock Market (U.S.)
Nasdaq Biotech Index
The following graph compares the cumulative
total stockholder return with the cumulative
total return of the Nasdaq Stock Market
(U.S.) and the Nasdaq Biotechnology Index,
assuming a $100 investment in BioMarin’s
common stock on December 31, 2002 and
reinvestment of dividends during the period.
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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
Stock Listing
BioMarin Pharmaceutical Inc.
is listed on the Nasdaq
Global Market and the
SWX Swiss Exchange under
the symbol BMRN.
Independent Accountants
KPMG LLP
San Francisco, CA
Mellon Investor Services LLC
480 Washington Boulevard
Jersey City, NJ 07310
Tel: 800-522-6645 (Domestic)
201-680-6578 (International)
Executives
Jean-Jacques Bienaimé
Chief Executive Officer
Jeffrey Cooper
Senior Vice President
Chief Financial Officer
Emil Kakkis, M.D., Ph.D.
Senior Vice President,
Chief Medical Officer
Stephen Aselage
Senior Vice President
Global Commercial Operations
Robert Baffi, Ph.D.
Senior Vice President
Technical Operations
Stuart Swiedler, M.D., Ph.D.
Senior Vice President
Clinical Affairs
Jeff Ajer
Vice President
Sales & Marketing Operations
William Aliski
Vice President &
General Manager
European Operations
R. Andrew Ramelmeier, Ph.D.
Vice President
Manufacturing and
Process Development
Victoria Sluzky, Ph.D.
Vice President
Quality & Analytical Chemistry
Amy Waterhouse
Vice President
Regulatory & Government Affairs
G. Eric Davis
Vice President
General Counsel & Secretary
Mark Wood
Vice President
Human Resources
Steven Jungles
Vice President
Supply Chain
Daniel Maher
Vice President
Product Development
Board of Directors
Jean-Jacques Bienaimé
Chief Executive Officer
BioMarin Pharmaceutical Inc.
Joseph Klein, III
Managing Director
Gauss Capital Advisors, LLC
Pierre Lapalme
Former President &
Chief Executive Officer
North America Ethypharm, Inc.
Michael Grey
President &
Chief Executive Officer
SGX Pharmaceuticals, Inc.
Alan Lewis, Ph.D.
President &
Chief Executive Officer
Novocell, Inc.
Elaine Heron, Ph.D.
Chairman &
Chief Executive Officer
Labcyte Inc.
Randy Meier
Executive Vice President,
Eye Care Business and
Operations, and
Chief Financial Officer
Advanced Medical Optics, Inc.
Forward-Looking Statement: This Annual Report contains ‘forward-looking statements’ as defined under securities laws.
These statements can generally be identified 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 could differ significantly 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 can-
didates, 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 influence 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 reflect later
events or circumstances or to reflect the occurrence of unanticipated events.
Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC. Naglazyme® and BioMarin are registered trademarks of
BioMarin Pharmaceutical Inc.
Jean-Jacques Bienaimé
Chief Executive Officer
Jeffrey Cooper
Senior Vice President,
Chief Financial Officer
Emil Kakkis, M.D., Ph.D.
Senior Vice President,
Chief Medical Officer
Stephen Aselage
Senior Vice President,
Global Commercial Operations
Robert Baffi, Ph.D.
Senior Vice President,
Technical Operations
Stuart Swiedler, M.D., Ph.D.
Senior Vice President,
Clinical Affairs
G. Eric Davis
Vice President,
General Counsel & Secretary
Mark Wood
Vice President,
Human Resources
Phenylase Core Team
Top row, left to right: James Dickow
(Director, Cell Culture), Gia DePillis, Ph.D.
(Director, Regulatory Affairs),
Laurie Tsuruda, Ph.D., D.A.B.T.
(Associate Director, Pharmacology/
Toxicology), Marlyn Morimoto, MS
(Sr. Financial Analyst), Paul Fitzpatrick,
Ph.D. (Principal Scientist, Research and
Development), Mubarack Muthalif, Ph.D.,
MBA (Senior Program Manager, Product
Development/ Phenylase Core Team
Leader) and Julie Wilson, Ph.D.
(Senior Product Manager).
In the Laboratory
Second row, left to right:
Carroll Henschell (Study Monitor,
Pharmacology/Toxicology) and
Sean Bell, Ph.D.
(Scientist, Research & Development).
Kuvan Core Team
Third row, left to right: Mark Henderson,
Ph.D. (Associate Director, Quality
Assurance), Alejandro Dorenbaum. M.D.
(Sr. Medical Director), Benjamin Dewees,
RAC (Senior Manager, Regulatory Affairs),
Julie Schraeder (Director, Finance),
Dong Wei, Ph.D. (Senior Program
Manager, Product Development),
Dan Oppenheimer, Ph.D. (Senior Director,
Program Management / Kuvan Program
Leader), Sandra Shpilberg, MBA (Director
of Marketing), V. Miles Rios, Jr., BA, MA
(Associate Director, Clinical Operations).
Representatives From
European Sales Team
Bottom row, left to right:
Philippe Carteron de Balmont (Country
Manager, Switzerland, France, &
BeNeLux), Guy Eggleton (Director of
Operations, BioMarin Europe Ltd.),
David Boothe, BSc (Director of Marketing,
BioMarin Europe) and Felis Iglesias
(Country Manager, Spain, Portugal).
Less than 10 years after opening our doors, we are approaching the antici-
pated launch of our third product. Our proven ability to deliver products to
market has transformed BioMarin from a product development-focused
company to a fully integrated commercial company with growing product
revenues and an exciting product pipeline. As important as our commercial
success, is the fact that our products are bringing the promise of new thera-
peutics to patients around the world suffering from rare genetic diseases.
Building A Meaningful Revenue Base
The efficiency and speed of our research, development and manufacturing
efforts is at the heart of our ability to quickly bring products to market. We
have successfully advanced each of our two flagship drugs, Aldurazyme®
(galsulfase) and Naglazyme® (laronidase), from research to commercializa-
tion in about five years. Revenues for both products increased substan-
tially in 2006, as we further expanded our reach to patients and secured
regulatory approvals worldwide. This sales growth, coupled with substan-
tial improvements to our capital structure, has significantly improved the
company’s financial profile.
2006 marked important progress in the commercialization efforts of
Aldurazyme. By the end of the year, worldwide sales increased 26 percent
to $96.3 million and as a result, BioMarin’s share of the profit in BioMarin/
Genzyme LLC increased 63.6% over fiscal 2005.
In 2006, Naglazyme, our first independently launched therapy for MPS VI,
was introduced in Europe after launching in the U.S. in mid-2005. When
considered as a stand-alone business, this product became profitable in
its first year on the market, and with $46.5 million in sales, we have clearly
established our ability to independently launch products for rare diseases.
This success now positions BioMarin as a commercial partner of choice for
marketing highly-specialized pharmaceutical products addressing the needs
of patients with rare diseases.
Expanding Product Profile
Growing revenues generated by Aldurazyme and Naglazyme are helping
us advance additional compounds in our product pipeline that target both
orphan genetic diseases and other conditions such as cardiovascular dis-
ease. This will present substantially larger market opportunities to augment
our current enzyme replacement franchise.
KuvanTM (sapropterin dihydrochloride), a small molecule oral therapeutic for
the treatment of phenylketonuria (PKU), has demonstrated strong safety
and efficacy in Phase 3 clinical trials and has been granted Fast Track status
by the FDA. This product, co-developed with Merck Serono, will be the
first approved drug treatment available for PKU, a metabolic disease that
affects more than 50,000 people in the developed world. Pending prior-
ity review designation and a favorable regulatory review, U.S. approval for
Kuvan is expected in late 2007. It will be the third drug we have success-
fully advanced from research to commercialization in less than five years.
The same enzyme cofactor found in Kuvan, 6R-BH4, has also been found
to play a key protective role in the cardiovascular system. To leverage this
asset in markets beyond PKU, we are conducting several proof-of-concept
studies for a variety of cardiovascular indications.
Finally, 2006 marked significant progress in our preclinical development of
PhenylaseTM, an enzyme substitution therapy for the treatment of severe
forms of PKU. We are currently performing additional preclinical studies with
the goal of filing an IND by the end of 2007 and initiating clinical studies in
early 2008.
Looking Forward
We are proud of our track record of bringing innovative, high-value biophar-
maceuticals to market. By leveraging clear-cut development strategy and
efficient, adaptive clinical development models we have achieved regula-
tory approvals far faster than the industry average and the results of our
progress are now becoming more tangible in the form of improved financial
performance. 2006 was a year of growth and progress in our efforts to
provide increased value to both investors and patients.
I would like to thank all of our employees and partners for their hard work
and commitment to excellence. We appreciate your continued support of
the company and look forward to keeping you informed of our progress
throughout the year.
Sincerely,
Jean-Jacques Bienaimé
Chief Executive Officer
2006 Milestones
Commercial Success
BioMarin has transformed from a product
development-focused company to a fully
integrated commercial company with
rapidly growing product revenues and an
exciting product pipeline.
Global Expansion
In 2006, BioMarin further established its
commercial presence in the United States
and Europe and is now expanding into
Latin America.
Improved Financial Profile
2006 was marked by significant increases
in product sales, a decrease in net loss,
the conversion of debt to common stock,
and an increasing cash balance. This has
enabled BioMarin to increase funding of
growth opportunities.
Proven Business Model
After less than four years in develop-
ment, BioMarin’s third product, Kuvan, is
expected to be approved in the U.S. by
the end of 2007, further demonstrating the
company’s ability to recognize unique
opportunities and develop them quickly.
Expanding Market Opportunities
BioMarin is now evaluating 6R-BH4 for
a variety of cardiovascular indications.
If successful, these programs could
significantly expand the company’s global
market potential.
Fulfilling Unmet Needs
BioMarin is bringing the promise of new
therapeutics to thousands of patients
worldwide who might otherwise go
untreated. In less than 10 years, the
company has developed and commer-
cialized two breakthrough drugs for rare
genetic diseases and is well positioned
to address many others with its exciting
product pipeline.
BioMarin Pharmaceutical Inc.
105 Digital Drive
Novato, CA 94949
Tel: 415.506.6700 415.
Fax: 415.382.7889
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BRINGING THEPROMISETOMARKET
CORPORATE INFORMATION
EXPERIENCED MANAGEMENT
A MESSAGE FROM THE CEO
2006 Annual Report
Stock Performance
BioMarin develops and commercializes
innovative biopharmaceuticals for serious
diseases and medical conditions. The
company’s portfolio comprises two
approved products and multiple clinical
and preclinical product candidates.
Corporate Headquarters
Transfer Agent
BioMarin
Nasdaq Stock Market (U.S.)
Nasdaq Biotech Index
The following graph compares the cumulative
total stockholder return with the cumulative
total return of the Nasdaq Stock Market
(U.S.) and the Nasdaq Biotechnology Index,
assuming a $100 investment in BioMarin’s
common stock on December 31, 2002 and
reinvestment of dividends during the period.
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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
Stock Listing
BioMarin Pharmaceutical Inc.
is listed on the Nasdaq
Global Market and the
SWX Swiss Exchange under
the symbol BMRN.
Independent Accountants
KPMG LLP
San Francisco, CA
Mellon Investor Services LLC
480 Washington Boulevard
Jersey City, NJ 07310
Tel: 800-522-6645 (Domestic)
201-680-6578 (International)
Executives
Jean-Jacques Bienaimé
Chief Executive Officer
Jeffrey Cooper
Senior Vice President
Chief Financial Officer
Emil Kakkis, M.D., Ph.D.
Senior Vice President,
Chief Medical Officer
Stephen Aselage
Senior Vice President
Global Commercial Operations
Robert Baffi, Ph.D.
Senior Vice President
Technical Operations
Stuart Swiedler, M.D., Ph.D.
Senior Vice President
Clinical Affairs
Jeff Ajer
Vice President
Sales & Marketing Operations
William Aliski
Vice President &
General Manager
European Operations
R. Andrew Ramelmeier, Ph.D.
Vice President
Manufacturing and
Process Development
Victoria Sluzky, Ph.D.
Vice President
Quality & Analytical Chemistry
Amy Waterhouse
Vice President
Regulatory & Government Affairs
G. Eric Davis
Vice President
General Counsel & Secretary
Mark Wood
Vice President
Human Resources
Steven Jungles
Vice President
Supply Chain
Daniel Maher
Vice President
Product Development
Board of Directors
Jean-Jacques Bienaimé
Chief Executive Officer
BioMarin Pharmaceutical Inc.
Joseph Klein, III
Managing Director
Gauss Capital Advisors, LLC
Pierre Lapalme
Former President &
Chief Executive Officer
North America Ethypharm, Inc.
Michael Grey
President &
Chief Executive Officer
SGX Pharmaceuticals, Inc.
Alan Lewis, Ph.D.
President &
Chief Executive Officer
Novocell, Inc.
Elaine Heron, Ph.D.
Chairman &
Chief Executive Officer
Labcyte Inc.
Randy Meier
Executive Vice President,
Eye Care Business and
Operations, and
Chief Financial Officer
Advanced Medical Optics, Inc.
Forward-Looking Statement: This Annual Report contains ‘forward-looking statements’ as defined under securities laws.
These statements can generally be identified 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 could differ significantly 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 can-
didates, 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 influence 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 reflect later
events or circumstances or to reflect the occurrence of unanticipated events.
Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC. Naglazyme® and BioMarin are registered trademarks of
BioMarin Pharmaceutical Inc.
Jean-Jacques Bienaimé
Chief Executive Officer
Jeffrey Cooper
Senior Vice President,
Chief Financial Officer
Emil Kakkis, M.D., Ph.D.
Senior Vice President,
Chief Medical Officer
Stephen Aselage
Senior Vice President,
Global Commercial Operations
Robert Baffi, Ph.D.
Senior Vice President,
Technical Operations
Stuart Swiedler, M.D., Ph.D.
Senior Vice President,
Clinical Affairs
G. Eric Davis
Vice President,
General Counsel & Secretary
Mark Wood
Vice President,
Human Resources
Phenylase Core Team
Top row, left to right: James Dickow
(Director, Cell Culture), Gia DePillis, Ph.D.
(Director, Regulatory Affairs),
Laurie Tsuruda, Ph.D., D.A.B.T.
(Associate Director, Pharmacology/
Toxicology), Marlyn Morimoto, MS
(Sr. Financial Analyst), Paul Fitzpatrick,
Ph.D. (Principal Scientist, Research and
Development), Mubarack Muthalif, Ph.D.,
MBA (Senior Program Manager, Product
Development/ Phenylase Core Team
Leader) and Julie Wilson, Ph.D.
(Senior Product Manager).
In the Laboratory
Second row, left to right:
Carroll Henschell (Study Monitor,
Pharmacology/Toxicology) and
Sean Bell, Ph.D.
(Scientist, Research & Development).
Kuvan Core Team
Third row, left to right: Mark Henderson,
Ph.D. (Associate Director, Quality
Assurance), Alejandro Dorenbaum. M.D.
(Sr. Medical Director), Benjamin Dewees,
RAC (Senior Manager, Regulatory Affairs),
Julie Schraeder (Director, Finance),
Dong Wei, Ph.D. (Senior Program
Manager, Product Development),
Dan Oppenheimer, Ph.D. (Senior Director,
Program Management / Kuvan Program
Leader), Sandra Shpilberg, MBA (Director
of Marketing), V. Miles Rios, Jr., BA, MA
(Associate Director, Clinical Operations).
Representatives From
European Sales Team
Bottom row, left to right:
Philippe Carteron de Balmont (Country
Manager, Switzerland, France, &
BeNeLux), Guy Eggleton (Director of
Operations, BioMarin Europe Ltd.),
David Boothe, BSc (Director of Marketing,
BioMarin Europe) and Felis Iglesias
(Country Manager, Spain, Portugal).
Less than 10 years after opening our doors, we are approaching the antici-
pated launch of our third product. Our proven ability to deliver products to
market has transformed BioMarin from a product development-focused
company to a fully integrated commercial company with growing product
revenues and an exciting product pipeline. As important as our commercial
success, is the fact that our products are bringing the promise of new thera-
peutics to patients around the world suffering from rare genetic diseases.
Building A Meaningful Revenue Base
The efficiency and speed of our research, development and manufacturing
efforts is at the heart of our ability to quickly bring products to market. We
have successfully advanced each of our two flagship drugs, Aldurazyme®
(laronidase) and Naglazyme® (galsulfase), from research to commercializa-
tion in about five years. Revenues for both products increased substan-
tially in 2006, as we further expanded our reach to patients and secured
regulatory approvals worldwide. This sales growth, coupled with substan-
tial improvements to our capital structure, has significantly improved the
company’s financial profile.
2006 marked important progress in the commercialization efforts of
Aldurazyme. By the end of the year, worldwide sales increased 26 percent
to $96.3 million and as a result, BioMarin’s share of the profit in BioMarin/
Genzyme LLC increased 63.6% over fiscal 2005.
In 2006, Naglazyme, our first independently launched therapy for MPS VI,
was introduced in Europe after launching in the U.S. in mid-2005. When
considered as a stand-alone business, this product became profitable in
its first year on the market, and with $46.5 million in sales, we have clearly
established our ability to independently launch products for rare diseases.
This success now positions BioMarin as a commercial partner of choice for
marketing highly-specialized pharmaceutical products addressing the needs
of patients with rare diseases.
Expanding Product Profile
Growing revenues generated by Aldurazyme and Naglazyme are helping
us advance additional compounds in our product pipeline that target both
orphan genetic diseases and other conditions such as cardiovascular dis-
ease. This will present substantially larger market opportunities to augment
our current enzyme replacement franchise.
KuvanTM (sapropterin dihydrochloride), a small molecule oral therapeutic for
the treatment of phenylketonuria (PKU), has demonstrated strong safety
and efficacy in Phase 3 clinical trials and has been granted Fast Track status
by the FDA. This product, co-developed with Merck Serono, will be the
first approved drug treatment available for PKU, a metabolic disease that
affects more than 50,000 people in the developed world. Pending prior-
ity review designation and a favorable regulatory review, U.S. approval for
Kuvan is expected in late 2007. It will be the third drug we have success-
fully advanced from research to commercialization in less than five years.
The same enzyme cofactor found in Kuvan, 6R-BH4, has also been found
to play a key protective role in the cardiovascular system. To leverage this
asset in markets beyond PKU, we are conducting several proof-of-concept
studies for a variety of cardiovascular indications.
Finally, 2006 marked significant progress in our preclinical development of
PhenylaseTM, an enzyme substitution therapy for the treatment of severe
forms of PKU. We are currently performing additional preclinical studies with
the goal of filing an IND by the end of 2007 and initiating clinical studies in
early 2008.
Looking Forward
We are proud of our track record of bringing innovative, high-value biophar-
maceuticals to market. By leveraging clear-cut development strategy and
efficient, adaptive clinical development models we have achieved regula-
tory approvals far faster than the industry average and the results of our
progress are now becoming more tangible in the form of improved financial
performance. 2006 was a year of growth and progress in our efforts to
provide increased value to both investors and patients.
I would like to thank all of our employees and partners for their hard work
and commitment to excellence. We appreciate your continued support of
the company and look forward to keeping you informed of our progress
throughout the year.
Sincerely,
Jean-Jacques Bienaimé
Chief Executive Officer
2006 Milestones
Commercial Success
BioMarin has transformed from a product
development-focused company to a fully
integrated commercial company with
rapidly growing product revenues and an
exciting product pipeline.
Global Expansion
In 2006, BioMarin further established its
commercial presence in the United States
and Europe and is now expanding into
Latin America.
Improved Financial Profile
2006 was marked by significant increases
in product sales, a decrease in net loss,
the conversion of debt to common stock,
and an increasing cash balance. This has
enabled BioMarin to increase funding of
growth opportunities.
Proven Business Model
After less than four years in develop-
ment, BioMarin’s third product, Kuvan, is
expected to be approved in the U.S. by
the end of 2007, further demonstrating the
company’s ability to recognize unique
opportunities and develop them quickly.
Expanding Market Opportunities
BioMarin is now evaluating 6R-BH4 for
a variety of cardiovascular indications.
If successful, these programs could
significantly expand the company’s global
market potential.
Fulfilling Unmet Needs
BioMarin is bringing the promise of new
therapeutics to thousands of patients
worldwide who might otherwise go
untreated. In less than 10 years, the
company has developed and commer-
cialized two breakthrough drugs for rare
genetic diseases and is well positioned
to address many others with its exciting
product pipeline.
BioMarin Pharmaceutical Inc.
105 Digital Drive
Novato, CA 94949
Tel: 415.506.6700 415.
Fax: 415.382.7889
i
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o
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BRINGING THEPROMISETOMARKET
CORPORATE INFORMATION
EXPERIENCED MANAGEMENT
A MESSAGE FROM THE CEO
2006 Annual Report
Stock Performance
BioMarin develops and commercializes
innovative biopharmaceuticals for serious
diseases and medical conditions. The
company’s portfolio comprises two
approved products and multiple clinical
and preclinical product candidates.
Corporate Headquarters
Transfer Agent
BioMarin
Nasdaq Stock Market (U.S.)
Nasdaq Biotech Index
The following graph compares the cumulative
total stockholder return with the cumulative
total return of the Nasdaq Stock Market
(U.S.) and the Nasdaq Biotechnology Index,
assuming a $100 investment in BioMarin’s
common stock on December 31, 2002 and
reinvestment of dividends during the period.
���
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����
����
����
����
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
Stock Listing
BioMarin Pharmaceutical Inc.
is listed on the Nasdaq
Global Market and the
SWX Swiss Exchange under
the symbol BMRN.
Independent Accountants
KPMG LLP
San Francisco, CA
Mellon Investor Services LLC
480 Washington Boulevard
Jersey City, NJ 07310
Tel: 800-522-6645 (Domestic)
201-680-6578 (International)
Executives
Jean-Jacques Bienaimé
Chief Executive Officer
Jeffrey Cooper
Senior Vice President
Chief Financial Officer
Emil Kakkis, M.D., Ph.D.
Senior Vice President,
Chief Medical Officer
Stephen Aselage
Senior Vice President
Global Commercial Operations
Robert Baffi, Ph.D.
Senior Vice President
Technical Operations
Stuart Swiedler, M.D., Ph.D.
Senior Vice President
Clinical Affairs
Jeff Ajer
Vice President
Sales & Marketing Operations
William Aliski
Vice President &
General Manager
European Operations
R. Andrew Ramelmeier, Ph.D.
Vice President
Manufacturing and
Process Development
Victoria Sluzky, Ph.D.
Vice President
Quality & Analytical Chemistry
Amy Waterhouse
Vice President
Regulatory & Government Affairs
G. Eric Davis
Vice President
General Counsel & Secretary
Mark Wood
Vice President
Human Resources
Steven Jungles
Vice President
Supply Chain
Daniel Maher
Vice President
Product Development
Board of Directors
Jean-Jacques Bienaimé
Chief Executive Officer
BioMarin Pharmaceutical Inc.
Joseph Klein, III
Managing Director
Gauss Capital Advisors, LLC
Pierre Lapalme
Former President &
Chief Executive Officer
North America Ethypharm, Inc.
Michael Grey
President &
Chief Executive Officer
SGX Pharmaceuticals, Inc.
Alan Lewis, Ph.D.
President &
Chief Executive Officer
Novocell, Inc.
Elaine Heron, Ph.D.
Chairman &
Chief Executive Officer
Labcyte Inc.
Randy Meier
Executive Vice President,
Eye Care Business and
Operations, and
Chief Financial Officer
Advanced Medical Optics, Inc.
Forward-Looking Statement: This Annual Report contains ‘forward-looking statements’ as defined under securities laws.
These statements can generally be identified 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 could differ significantly 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 can-
didates, 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 influence 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 reflect later
events or circumstances or to reflect the occurrence of unanticipated events.
Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC. Naglazyme® and BioMarin are registered trademarks of
BioMarin Pharmaceutical Inc.
Jean-Jacques Bienaimé
Chief Executive Officer
Jeffrey Cooper
Senior Vice President,
Chief Financial Officer
Emil Kakkis, M.D., Ph.D.
Senior Vice President,
Chief Medical Officer
Stephen Aselage
Senior Vice President,
Global Commercial Operations
Robert Baffi, Ph.D.
Senior Vice President,
Technical Operations
Stuart Swiedler, M.D., Ph.D.
Senior Vice President,
Clinical Affairs
G. Eric Davis
Vice President,
General Counsel & Secretary
Mark Wood
Vice President,
Human Resources
Phenylase Core Team
Top row, left to right: James Dickow
(Director, Cell Culture), Gia DePillis, Ph.D.
(Director, Regulatory Affairs),
Laurie Tsuruda, Ph.D., D.A.B.T.
(Associate Director, Pharmacology/
Toxicology), Marlyn Morimoto, MS
(Sr. Financial Analyst), Paul Fitzpatrick,
Ph.D. (Principal Scientist, Research and
Development), Mubarack Muthalif, Ph.D.,
MBA (Senior Program Manager, Product
Development/ Phenylase Core Team
Leader) and Julie Wilson, Ph.D.
(Senior Product Manager).
In the Laboratory
Second row, left to right:
Carroll Henschell (Study Monitor,
Pharmacology/Toxicology) and
Sean Bell, Ph.D.
(Scientist, Research & Development).
Kuvan Core Team
Third row, left to right: Mark Henderson,
Ph.D. (Associate Director, Quality
Assurance), Alejandro Dorenbaum. M.D.
(Sr. Medical Director), Benjamin Dewees,
RAC (Senior Manager, Regulatory Affairs),
Julie Schraeder (Director, Finance),
Dong Wei, Ph.D. (Senior Program
Manager, Product Development),
Dan Oppenheimer, Ph.D. (Senior Director,
Program Management / Kuvan Program
Leader), Sandra Shpilberg, MBA (Director
of Marketing), V. Miles Rios, Jr., BA, MA
(Associate Director, Clinical Operations).
Representatives From
European Sales Team
Bottom row, left to right:
Philippe Carteron de Balmont (Country
Manager, Switzerland, France, &
BeNeLux), Guy Eggleton (Director of
Operations, BioMarin Europe Ltd.),
David Boothe, BSc (Director of Marketing,
BioMarin Europe) and Felis Iglesias
(Country Manager, Spain, Portugal).
Less than 10 years after opening our doors, we are approaching the antici-
pated launch of our third product. Our proven ability to deliver products to
market has transformed BioMarin from a product development-focused
company to a fully integrated commercial company with growing product
revenues and an exciting product pipeline. As important as our commercial
success, is the fact that our products are bringing the promise of new thera-
peutics to patients around the world suffering from rare genetic diseases.
Building A Meaningful Revenue Base
The efficiency and speed of our research, development and manufacturing
efforts is at the heart of our ability to quickly bring products to market. We
have successfully advanced each of our two flagship drugs, Aldurazyme®
(galsulfase) and Naglazyme® (laronidase), from research to commercializa-
tion in about five years. Revenues for both products increased substan-
tially in 2006, as we further expanded our reach to patients and secured
regulatory approvals worldwide. This sales growth, coupled with substan-
tial improvements to our capital structure, has significantly improved the
company’s financial profile.
2006 marked important progress in the commercialization efforts of
Aldurazyme. By the end of the year, worldwide sales increased 26 percent
to $96.3 million and as a result, BioMarin’s share of the profit in BioMarin/
Genzyme LLC increased 63.6% over fiscal 2005.
In 2006, Naglazyme, our first independently launched therapy for MPS VI,
was introduced in Europe after launching in the U.S. in mid-2005. When
considered as a stand-alone business, this product became profitable in
its first year on the market, and with $46.5 million in sales, we have clearly
established our ability to independently launch products for rare diseases.
This success now positions BioMarin as a commercial partner of choice for
marketing highly-specialized pharmaceutical products addressing the needs
of patients with rare diseases.
Expanding Product Profile
Growing revenues generated by Aldurazyme and Naglazyme are helping
us advance additional compounds in our product pipeline that target both
orphan genetic diseases and other conditions such as cardiovascular dis-
ease. This will present substantially larger market opportunities to augment
our current enzyme replacement franchise.
KuvanTM (sapropterin dihydrochloride), a small molecule oral therapeutic for
the treatment of phenylketonuria (PKU), has demonstrated strong safety
and efficacy in Phase 3 clinical trials and has been granted Fast Track status
by the FDA. This product, co-developed with Merck Serono, will be the
first approved drug treatment available for PKU, a metabolic disease that
affects more than 50,000 people in the developed world. Pending prior-
ity review designation and a favorable regulatory review, U.S. approval for
Kuvan is expected in late 2007. It will be the third drug we have success-
fully advanced from research to commercialization in less than five years.
The same enzyme cofactor found in Kuvan, 6R-BH4, has also been found
to play a key protective role in the cardiovascular system. To leverage this
asset in markets beyond PKU, we are conducting several proof-of-concept
studies for a variety of cardiovascular indications.
Finally, 2006 marked significant progress in our preclinical development of
PhenylaseTM, an enzyme substitution therapy for the treatment of severe
forms of PKU. We are currently performing additional preclinical studies with
the goal of filing an IND by the end of 2007 and initiating clinical studies in
early 2008.
Looking Forward
We are proud of our track record of bringing innovative, high-value biophar-
maceuticals to market. By leveraging clear-cut development strategy and
efficient, adaptive clinical development models we have achieved regula-
tory approvals far faster than the industry average and the results of our
progress are now becoming more tangible in the form of improved financial
performance. 2006 was a year of growth and progress in our efforts to
provide increased value to both investors and patients.
I would like to thank all of our employees and partners for their hard work
and commitment to excellence. We appreciate your continued support of
the company and look forward to keeping you informed of our progress
throughout the year.
Sincerely,
Jean-Jacques Bienaimé
Chief Executive Officer
2006 Milestones
Commercial Success
BioMarin has transformed from a product
development-focused company to a fully
integrated commercial company with
rapidly growing product revenues and an
exciting product pipeline.
Global Expansion
In 2006, BioMarin further established its
commercial presence in the United States
and Europe and is now expanding into
Latin America.
Improved Financial Profile
2006 was marked by significant increases
in product sales, a decrease in net loss,
the conversion of debt to common stock,
and an increasing cash balance. This has
enabled BioMarin to increase funding of
growth opportunities.
Proven Business Model
After less than four years in develop-
ment, BioMarin’s third product, Kuvan, is
expected to be approved in the U.S. by
the end of 2007, further demonstrating the
company’s ability to recognize unique
opportunities and develop them quickly.
Expanding Market Opportunities
BioMarin is now evaluating 6R-BH4 for
a variety of cardiovascular indications.
If successful, these programs could
significantly expand the company’s global
market potential.
Fulfilling Unmet Needs
BioMarin is bringing the promise of new
therapeutics to thousands of patients
worldwide who might otherwise go
untreated. In less than 10 years, the
company has developed and commer-
cialized two breakthrough drugs for rare
genetic diseases and is well positioned
to address many others with its exciting
product pipeline.
BioMarin Pharmaceutical Inc.
105 Digital Drive
Novato, CA 94949
Tel: 415.506.6700 415.
Fax: 415.382.7889
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BRINGING THEPROMISETOMARKET
Preclinical
Phase I
Phase II
Phase III
BLA / NDA / MAA
On The Market
NAGLAZYME® for mucopolysaccharidosis VI (MPS VI)
MPS VI is a rare, inherited metabolic disease
caused by a deficiency in arylsulfatase B, an
enzyme involved in the carbohydrate recycling
throughout the body. Over time, the disease
progresses, causing severe disabilities such
as impaired cardiac and pulmonary function,
delayed physical development, skeletal and joint
deformities, reduced endurance and impaired
vision and hearing. The majority of untreated
patients die from disease-related complications
between childhood and early adulthood.
Naglazyme is the first drug approved to treat
MPS VI in the United States and Europe. It is an
enzyme replacement therapy, using a recombinant
version of arylsulfatase B to replace or supplement
low to non-existent levels of the natural enzyme
in the body.
Phase 1 clinical trials of
Naglazyme were completed
in 2001, demonstrating product
safety and early indications
of efficacy.
A Phase 2 open-label study of Naglazyme was
conducted in the U.S. and Australia in 2002
evaluating the safety and efficacy of the drug in
subjects ranging in age from six to 22. Long-term
results showed patients improved their walk
distances over baseline and stair-climbing ability.
Functional improvements were also observed.
Conducted at six international sites, the Phase 3
trial of Naglazyme was completed in early 2004.
The trial enrolled patients ranging in age from five
to 29 years old and ran for 24 consecutive weeks.
Patients in this study also showed statistically
significant improvement in measurements of endur-
ance, including walk distances and stair climbing.
Strong safety and efficacy data generated in the
Naglazyme clinical trials formed the basis of
BioMarin’s U.S. and European regulatory submis-
sions in late 2004. In all, 56 patients participated
in the trials in seven different countries.
As Naglazyme moved into Phase 3 clinical trials,
the company expanded its manufacturing facility
to meet growing needs and product demand. The
product received FDA approval in May 2005 and
EMEA approval in January 2006, making it the first
approved drug treatment for MPS VI.
Naglazyme was launched in the U.S. in June
2005 and in the EU in February 2006. In 2007
and beyond, growth will be driven by factors
such as new patient identification, geographic
expansion and increasing dosage requirements.
Naglazyme Revenue by Quarters
(dollars in millions)
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ALDURAZYME® for mucopolysaccharidosis I (MPS I)
Like MPS VI, MPS I is a rare, inherited metabolic
disease caused by a deficiency of alpha-L-
iduronidase, an enzyme involved in the
carbohydrate recycling throughout the body.
Symptoms can include impaired cardiac and
pulmonary function, delayed physical develop-
ment, skeletal and joint deformities, reduced
endurance, and delayed mental function.
Untreated patients can die from complications
before adulthood.
Aldurazyme is the first drug approved to treat MPS I
in the United States and Europe, and was recently
approved for use in Japan. It is an enzyme replace-
ment therapy, using a recombinant version of alpha-
L-iduronidase to replace or supplement low to non-
existent levels of the natural enzyme in the body.
KUVAN™ for PKU
Phenylketonuria (PKU) is a rare, inherited
metabolic disease resulting from a deficiency
of phenylalanine hydroxylase, the enzyme
responsible for converting phenylalanine (Phe)
to tyrosine. Sustained elevated blood Phe
levels can result in serious and irreversible
neurological damage. In all countries where
modern medical technology is available, all
newborns are screened for PKU. There are
approximately 50,000 PKU patients under the
age of 40 in the developed world.
Kuvan (sapropterin dihydrochloride), an oral small
molecule therapeutic, is the synthetic form of
6R-BH4 (tetrahydrobiopterin), a naturally-occurring
enzyme cofactor that works in conjunction with
phenylalanine hydroxylase to metabolize Phe.
6R-BH4 for cardiovascular disease and sickle cell disease
6R-BH4 (tetrahydrobiopterin), more commonly
known as BH4, is a naturally-occurring enzyme
cofactor that is required for numerous biochemi-
cal and physiologic processes, including the
synthesis of nitric oxide (NO). NO has been
shown to play a key protective role throughout
the cardiovascular system.
A deficiency of BH4 can disrupt the production of
endothelial NO, leading to endothelial dysfunction,
which has been associated with many cardiovascu-
lar diseases impacting millions of people worldwide.
PHENYLASE™ for PKU
BioMarin expects to file an IND application for
Phenylase in late 2007. Phenylase is an investi-
gational enzyme substitution therapy designed
to treat severe PKU, specifically in patients who
are non-BH4 responsive. The active ingredient,
phenylalanine ammonia lyase, is designed to
break down Phe that builds up due to lack of or
diminished activity of the enzyme phenylalanine
hydroxylase.
Study shows weekly treatment of Phenylase has
substantial impact on Phe levels in PKU mice.
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In 1998, BioMarin and Genzyme formed BioMarin/
Genzyme LLC to develop and commercialize
Aldurazyme worldwide. Early trials demonstrated
safety and promising clinical results and in late
2001, a pivotal Phase 3 trial demonstrated a
statistically significant increase in patients’ pulmo-
nary capacity, as well as increased endurance.
An extension study followed 45 patients who
continued the treatment for an additional three
years in which they demonstrated further
improvement or stabilization in pulmonary
function and endurance.
With FDA and EC EMEA approval granted in
2003, Aldurazyme became the first commercially
available treatment for MPS I, and provided
physicians with the ability to change the course
of the disease and provide better outcomes
for patients worldwide.
BioMarin’s strategic partner, Merck Serono, is
supporting the development of Kuvan and has
rights to market the product outside of the U.S.
and Japan. BioMarin retains exclusive rights to
market Kuvan in the United States.
Data from Phase 3 clinical trials demonstrated
the safety and tolerability of Kuvan, as well as
its ability to increase Phe tolerance and reduce
blood Phe levels in PKU patients.
Approval for Kuvan is expected in late 2007 in
the U.S. and in 2008 in Europe.
BH4 has also shown promise as a
treatment for diseases where en-
dothelial dysfunction plays a larger
role. To identify potential indications,
BioMarin is conducting small, proof-
of-concept studies for a number of
cardiovascular diseases.
In the second quarter of 2007, the company
expects to initiate a Phase 2 clinical study in
sickle cell disease (SCD), an inherited blood
disorder that affects red blood cells. There are
an estimated 70,000 – 100,000 SCD patients
in the U.S.
In the second quarter of 2007, an investigator-
sponsored Phase 1 trial of BH4 in pulmonary
arterial hypertension (PAH) is expected to be ini-
tiated. PAH is a chronic, life-threatening disease
afflicting 100,000 to 200,000 people worldwide.
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Phase 3 Diet Study: Final Phe tolerance
of the Kuvan group increased, on average, to
half the amount of Phe in a normal diet.
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The product has been on the market since 2003
and has generated increased sales worldwide
through 2006.
Aldurazyme Annual Revenue
(dollars in millions)
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BioMarin’s market capitalization has more
than tripled over the last two years.
(dollars in millions)
������
����
����
����
����
����
BioMarin has transformed from
a product development-oriented
company to a fully integrated
commercial company with growing
product revenues and an exciting
product pipeline.
BioMarin products are bringing
the promise of new therapeutics to
patients around the world suffering
from rare genetic diseases.
BioMarin is now a commercial
partner of choice for marketing
highly-specialized pharmaceutical
products that target both
orphan diseases and more
common conditions such as
cardiovascular disease.
By leveraging clear-cut develop-
ment strategy and efficient, adap-
tive clinical development models,
BioMarin has achieved regulatory
approvals far faster than the
industry average.
Expected milestones in 2007–08
include the anticipated launch
of Kuvan, the initiation of clinical
studies of Phenylase and preclinical
studies of 6R-BH4 for a variety of
cardiovascular indications.
BioMarin’s two flagship drugs,
Aldurazyme® (galsulfase) for MPS I,
and Naglazyme® (laronidase) for
MPS VI, successfully advanced
from research to commercialization
status in about five years.
Naglazyme, BioMarin’s first
independently launched therapy,
became profitable in its first year
on the market. With full worldwide
rights, the company is now enjoying
commercial success in the United
States and Europe and is expand-
ing into Latin America.
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H
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HOHOHCCCHON NH NH NH H N 2 3 5678H
Preclinical
Phase I
Phase II
Phase III
BLA / NDA / MAA
On The Market
NAGLAZYME® for mucopolysaccharidosis VI (MPS VI)
MPS VI is a rare, inherited metabolic disease
caused by a deficiency in arylsulfatase B, an
enzyme involved in the carbohydrate recycling
throughout the body. Over time, the disease
progresses, causing severe disabilities such
as impaired cardiac and pulmonary function,
delayed physical development, skeletal and joint
deformities, reduced endurance and impaired
vision and hearing. The majority of untreated
patients die from disease-related complications
between childhood and early adulthood.
Naglazyme is the first drug approved to treat
MPS VI in the United States and Europe. It is an
enzyme replacement therapy, using a recombinant
version of arylsulfatase B to replace or supplement
low to non-existent levels of the natural enzyme
in the body.
Phase 1 clinical trials of
Naglazyme were completed
in 2001, demonstrating product
safety and early indications
of efficacy.
A Phase 2 open-label study of Naglazyme was
conducted in the U.S. and Australia in 2002
evaluating the safety and efficacy of the drug in
subjects ranging in age from six to 22. Long-term
results showed patients improved their walk
distances over baseline and stair-climbing ability.
Functional improvements were also observed.
Conducted at six international sites, the Phase 3
trial of Naglazyme was completed in early 2004.
The trial enrolled patients ranging in age from five
to 29 years old and ran for 24 consecutive weeks.
Patients in this study also showed statistically
significant improvement in measurements of endur-
ance, including walk distances and stair climbing.
Strong safety and efficacy data generated in the
Naglazyme clinical trials formed the basis of
BioMarin’s U.S. and European regulatory submis-
sions in late 2004. In all, 56 patients participated
in the trials in seven different countries.
As Naglazyme moved into Phase 3 clinical trials,
the company expanded its manufacturing facility
to meet growing needs and product demand. The
product received FDA approval in May 2005 and
EMEA approval in January 2006, making it the first
approved drug treatment for MPS VI.
Naglazyme was launched in the U.S. in June
2005 and in the EU in February 2006. In 2007
and beyond, growth will be driven by factors
such as new patient identification, geographic
expansion and increasing dosage requirements.
Naglazyme Revenue by Quarters
(dollars in millions)
�����
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�����
����
����
����
���� ���� ���� ���� ���� ����
ALDURAZYME® for mucopolysaccharidosis I (MPS I)
Like MPS VI, MPS I is a rare, inherited metabolic
disease caused by a deficiency of alpha-L-
iduronidase, an enzyme involved in the
carbohydrate recycling throughout the body.
Symptoms can include impaired cardiac and
pulmonary function, delayed physical develop-
ment, skeletal and joint deformities, reduced
endurance, and delayed mental function.
Untreated patients can die from complications
before adulthood.
Aldurazyme is the first drug approved to treat MPS I
in the United States and Europe, and was recently
approved for use in Japan. It is an enzyme replace-
ment therapy, using a recombinant version of alpha-
L-iduronidase to replace or supplement low to non-
existent levels of the natural enzyme in the body.
KUVAN™ for PKU
Phenylketonuria (PKU) is a rare, inherited
metabolic disease resulting from a deficiency
of phenylalanine hydroxylase, the enzyme
responsible for converting phenylalanine (Phe)
to tyrosine. Sustained elevated blood Phe
levels can result in serious and irreversible
neurological damage. In all countries where
modern medical technology is available, all
newborns are screened for PKU. There are
approximately 50,000 PKU patients under the
age of 40 in the developed world.
Kuvan (sapropterin dihydrochloride), an oral small
molecule therapeutic, is the synthetic form of
6R-BH4 (tetrahydrobiopterin), a naturally-occurring
enzyme cofactor that works in conjunction with
phenylalanine hydroxylase to metabolize Phe.
6R-BH4 for cardiovascular disease and sickle cell disease
6R-BH4 (tetrahydrobiopterin), more commonly
known as BH4, is a naturally-occurring enzyme
cofactor that is required for numerous biochemi-
cal and physiologic processes, including the
synthesis of nitric oxide (NO). NO has been
shown to play a key protective role throughout
the cardiovascular system.
A deficiency of BH4 can disrupt the production of
endothelial NO, leading to endothelial dysfunction,
which has been associated with many cardiovascu-
lar diseases impacting millions of people worldwide.
PHENYLASE™ for PKU
BioMarin expects to file an IND application for
Phenylase in late 2007. Phenylase is an investi-
gational enzyme substitution therapy designed
to treat severe PKU, specifically in patients who
are non-BH4 responsive. The active ingredient,
phenylalanine ammonia lyase, is designed to
break down Phe that builds up due to lack of or
diminished activity of the enzyme phenylalanine
hydroxylase.
Study shows weekly treatment of Phenylase has
substantial impact on Phe levels in PKU mice.
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In 1998, BioMarin and Genzyme formed BioMarin/
Genzyme LLC to develop and commercialize
Aldurazyme worldwide. Early trials demonstrated
safety and promising clinical results and in late
2001, a pivotal Phase 3 trial demonstrated a
statistically significant increase in patients’ pulmo-
nary capacity, as well as increased endurance.
An extension study followed 45 patients who
continued the treatment for an additional three
years in which they demonstrated further
improvement or stabilization in pulmonary
function and endurance.
With FDA and EC EMEA approval granted in
2003, Aldurazyme became the first commercially
available treatment for MPS I, and provided
physicians with the ability to change the course
of the disease and provide better outcomes
for patients worldwide.
BioMarin’s strategic partner, Merck Serono, is
supporting the development of Kuvan and has
rights to market the product outside of the U.S.
and Japan. BioMarin retains exclusive rights to
market Kuvan in the United States.
Data from Phase 3 clinical trials demonstrated
the safety and tolerability of Kuvan, as well as
its ability to increase Phe tolerance and reduce
blood Phe levels in PKU patients.
Approval for Kuvan is expected in late 2007 in
the U.S. and in 2008 in Europe.
BH4 has also shown promise as a
treatment for diseases where en-
dothelial dysfunction plays a larger
role. To identify potential indications,
BioMarin is conducting small, proof-
of-concept studies for a number of
cardiovascular diseases.
In the second quarter of 2007, the company
expects to initiate a Phase 2 clinical study in
sickle cell disease (SCD), an inherited blood
disorder that affects red blood cells. There are
an estimated 70,000 – 100,000 SCD patients
in the U.S.
In the second quarter of 2007, an investigator-
sponsored Phase 1 trial of BH4 in pulmonary
arterial hypertension (PAH) is expected to be ini-
tiated. PAH is a chronic, life-threatening disease
afflicting 100,000 to 200,000 people worldwide.
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Phase 3 Diet Study: Final Phe tolerance
of the Kuvan group increased, on average, to
half the amount of Phe in a normal diet.
�������
�����
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�
�
�
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����
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The product has been on the market since 2003
and has generated increased sales worldwide
through 2006.
Aldurazyme Annual Revenue
(dollars in millions)
�����
�����
�����
�����
����
����
����
����
BioMarin’s market capitalization has more
than tripled over the last two years.
(dollars in millions)
������
����
����
����
����
����
BioMarin has transformed from
a product development-oriented
company to a fully integrated
commercial company with growing
product revenues and an exciting
product pipeline.
BioMarin products are bringing
the promise of new therapeutics to
patients around the world suffering
from rare genetic diseases.
BioMarin is now a commercial
partner of choice for marketing
highly-specialized pharmaceutical
products that target both
orphan diseases and more
common conditions such as
cardiovascular disease.
By leveraging clear-cut develop-
ment strategy and efficient, adap-
tive clinical development models,
BioMarin has achieved regulatory
approvals far faster than the
industry average.
Expected milestones in 2007–08
include the anticipated launch
of Kuvan, the initiation of clinical
studies of Phenylase and preclinical
studies of 6R-BH4 for a variety of
cardiovascular indications.
BioMarin’s two flagship drugs,
Aldurazyme® (galsulfase) for MPS I,
and Naglazyme® (laronidase) for
MPS VI, successfully advanced
from research to commercialization
status in about five years.
Naglazyme, BioMarin’s first
independently launched therapy,
became profitable in its first year
on the market. With full worldwide
rights, the company is now enjoying
commercial success in the United
States and Europe and is expand-
ing into Latin America.
T
E
K
R
A
M
E
H
T
N
O
A
D
N
-
E
R
P
T
N
E
M
P
O
L
E
V
E
D
N
I
HOHOHCCCHON NH NH NH H N 2 3 5678H
Preclinical
Phase I
Phase II
Phase III
BLA / NDA / MAA
On The Market
NAGLAZYME® for mucopolysaccharidosis VI (MPS VI)
MPS VI is a rare, inherited metabolic disease
caused by a deficiency in arylsulfatase B, an
enzyme involved in the carbohydrate recycling
throughout the body. Over time, the disease
progresses, causing severe disabilities such
as impaired cardiac and pulmonary function,
delayed physical development, skeletal and joint
deformities, reduced endurance and impaired
vision and hearing. The majority of untreated
patients die from disease-related complications
between childhood and early adulthood.
Naglazyme is the first drug approved to treat
MPS VI in the United States and Europe. It is an
enzyme replacement therapy, using a recombinant
version of arylsulfatase B to replace or supplement
low to non-existent levels of the natural enzyme
in the body.
Phase 1 clinical trials of
Naglazyme were completed
in 2001, demonstrating product
safety and early indications
of efficacy.
A Phase 2 open-label study of Naglazyme was
conducted in the U.S. and Australia in 2002
evaluating the safety and efficacy of the drug in
subjects ranging in age from six to 22. Long-term
results showed patients improved their walk
distances over baseline and stair-climbing ability.
Functional improvements were also observed.
Conducted at six international sites, the Phase 3
trial of Naglazyme was completed in early 2004.
The trial enrolled patients ranging in age from five
to 29 years old and ran for 24 consecutive weeks.
Patients in this study also showed statistically
significant improvement in measurements of endur-
ance, including walk distances and stair climbing.
Strong safety and efficacy data generated in the
Naglazyme clinical trials formed the basis of
BioMarin’s U.S. and European regulatory submis-
sions in late 2004. In all, 56 patients participated
in the trials in seven different countries.
As Naglazyme moved into Phase 3 clinical trials,
the company expanded its manufacturing facility
to meet growing needs and product demand. The
product received FDA approval in May 2005 and
EMEA approval in January 2006, making it the first
approved drug treatment for MPS VI.
Naglazyme was launched in the U.S. in June
2005 and in the EU in February 2006. In 2007
and beyond, growth will be driven by factors
such as new patient identification, geographic
expansion and increasing dosage requirements.
Naglazyme Revenue by Quarters
(dollars in millions)
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ALDURAZYME® for mucopolysaccharidosis I (MPS I)
Like MPS VI, MPS I is a rare, inherited metabolic
disease caused by a deficiency of alpha-L-
iduronidase, an enzyme involved in the
carbohydrate recycling throughout the body.
Symptoms can include impaired cardiac and
pulmonary function, delayed physical develop-
ment, skeletal and joint deformities, reduced
endurance, and delayed mental function.
Untreated patients can die from complications
before adulthood.
Aldurazyme is the first drug approved to treat MPS I
in the United States and Europe, and was recently
approved for use in Japan. It is an enzyme replace-
ment therapy, using a recombinant version of alpha-
L-iduronidase to replace or supplement low to non-
existent levels of the natural enzyme in the body.
KUVAN™ for PKU
Phenylketonuria (PKU) is a rare, inherited
metabolic disease resulting from a deficiency
of phenylalanine hydroxylase, the enzyme
responsible for converting phenylalanine (Phe)
to tyrosine. Sustained elevated blood Phe
levels can result in serious and irreversible
neurological damage. In all countries where
modern medical technology is available, all
newborns are screened for PKU. There are
approximately 50,000 PKU patients under the
age of 40 in the developed world.
Kuvan (sapropterin dihydrochloride), an oral small
molecule therapeutic, is the synthetic form of
6R-BH4 (tetrahydrobiopterin), a naturally-occurring
enzyme cofactor that works in conjunction with
phenylalanine hydroxylase to metabolize Phe.
6R-BH4 for cardiovascular disease and sickle cell disease
6R-BH4 (tetrahydrobiopterin), more commonly
known as BH4, is a naturally-occurring enzyme
cofactor that is required for numerous biochemi-
cal and physiologic processes, including the
synthesis of nitric oxide (NO). NO has been
shown to play a key protective role throughout
the cardiovascular system.
A deficiency of BH4 can disrupt the production of
endothelial NO, leading to endothelial dysfunction,
which has been associated with many cardiovascu-
lar diseases impacting millions of people worldwide.
PHENYLASE™ for PKU
BioMarin expects to file an IND application for
Phenylase in late 2007. Phenylase is an investi-
gational enzyme substitution therapy designed
to treat severe PKU, specifically in patients who
are non-BH4 responsive. The active ingredient,
phenylalanine ammonia lyase, is designed to
break down Phe that builds up due to lack of or
diminished activity of the enzyme phenylalanine
hydroxylase.
Study shows weekly treatment of Phenylase has
substantial impact on Phe levels in PKU mice.
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In 1998, BioMarin and Genzyme formed BioMarin/
Genzyme LLC to develop and commercialize
Aldurazyme worldwide. Early trials demonstrated
safety and promising clinical results and in late
2001, a pivotal Phase 3 trial demonstrated a
statistically significant increase in patients’ pulmo-
nary capacity, as well as increased endurance.
An extension study followed 45 patients who
continued the treatment for an additional three
years in which they demonstrated further
improvement or stabilization in pulmonary
function and endurance.
With FDA and EC EMEA approval granted in
2003, Aldurazyme became the first commercially
available treatment for MPS I, and provided
physicians with the ability to change the course
of the disease and provide better outcomes
for patients worldwide.
BioMarin’s strategic partner, Merck Serono, is
supporting the development of Kuvan and has
rights to market the product outside of the U.S.
and Japan. BioMarin retains exclusive rights to
market Kuvan in the United States.
Data from Phase 3 clinical trials demonstrated
the safety and tolerability of Kuvan, as well as
its ability to increase Phe tolerance and reduce
blood Phe levels in PKU patients.
Approval for Kuvan is expected in late 2007 in
the U.S. and in 2008 in Europe.
BH4 has also shown promise as a
treatment for diseases where en-
dothelial dysfunction plays a larger
role. To identify potential indications,
BioMarin is conducting small, proof-
of-concept studies for a number of
cardiovascular diseases.
In the second quarter of 2007, the company
expects to initiate a Phase 2 clinical study in
sickle cell disease (SCD), an inherited blood
disorder that affects red blood cells. There are
an estimated 70,000 – 100,000 SCD patients
in the U.S.
In the second quarter of 2007, an investigator-
sponsored Phase 1 trial of BH4 in pulmonary
arterial hypertension (PAH) is expected to be ini-
tiated. PAH is a chronic, life-threatening disease
afflicting 100,000 to 200,000 people worldwide.
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Phase 3 Diet Study: Final Phe tolerance
of the Kuvan group increased, on average, to
half the amount of Phe in a normal diet.
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The product has been on the market since 2003
and has generated increased sales worldwide
through 2006.
Aldurazyme Annual Revenue
(dollars in millions)
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BioMarin’s market capitalization has more
than tripled over the last two years.
(dollars in millions)
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BioMarin has transformed from
a product development-oriented
company to a fully integrated
commercial company with growing
product revenues and an exciting
product pipeline.
BioMarin products are bringing
the promise of new therapeutics to
patients around the world suffering
from rare genetic diseases.
BioMarin is now a commercial
partner of choice for marketing
highly-specialized pharmaceutical
products that target both
orphan diseases and more
common conditions such as
cardiovascular disease.
By leveraging clear-cut develop-
ment strategy and efficient, adap-
tive clinical development models,
BioMarin has achieved regulatory
approvals far faster than the
industry average.
Expected milestones in 2007–08
include the anticipated launch
of Kuvan, the initiation of clinical
studies of Phenylase and preclinical
studies of 6R-BH4 for a variety of
cardiovascular indications.
BioMarin’s two flagship drugs,
Aldurazyme® (galsulfase) for MPS I,
and Naglazyme® (laronidase) for
MPS VI, successfully advanced
from research to commercialization
status in about five years.
Naglazyme, BioMarin’s first
independently launched therapy,
became profitable in its first year
on the market. With full worldwide
rights, the company is now enjoying
commercial success in the United
States and Europe and is expand-
ing into Latin America.
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HOHOHCCCHON NH NH NH H N 2 3 5678H
CORPORATE INFORMATION
EXPERIENCED MANAGEMENT
A MESSAGE FROM THE CEO
2006 Annual Report
Stock Performance
BioMarin develops and commercializes
innovative biopharmaceuticals for serious
diseases and medical conditions. The
company’s portfolio comprises two
approved products and multiple clinical
and preclinical product candidates.
Corporate Headquarters
Transfer Agent
BioMarin
Nasdaq Stock Market (U.S.)
Nasdaq Biotech Index
The following graph compares the cumulative
total stockholder return with the cumulative
total return of the Nasdaq Stock Market
(U.S.) and the Nasdaq Biotechnology Index,
assuming a $100 investment in BioMarin’s
common stock on December 31, 2002 and
reinvestment of dividends during the period.
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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
Stock Listing
BioMarin Pharmaceutical Inc.
is listed on the Nasdaq
Global Market and the
SWX Swiss Exchange under
the symbol BMRN.
Independent Accountants
KPMG LLP
San Francisco, CA
Mellon Investor Services LLC
480 Washington Boulevard
Jersey City, NJ 07310
Tel: 800-522-6645 (Domestic)
201-680-6578 (International)
Executives
Jean-Jacques Bienaimé
Chief Executive Officer
Jeffrey Cooper
Senior Vice President
Chief Financial Officer
Emil Kakkis, M.D., Ph.D.
Senior Vice President,
Chief Medical Officer
Stephen Aselage
Senior Vice President
Global Commercial Operations
Robert Baffi, Ph.D.
Senior Vice President
Technical Operations
Stuart Swiedler, M.D., Ph.D.
Senior Vice President
Clinical Affairs
Jeff Ajer
Vice President
Sales & Marketing Operations
William Aliski
Vice President &
General Manager
European Operations
R. Andrew Ramelmeier, Ph.D.
Vice President
Manufacturing and
Process Development
Victoria Sluzky, Ph.D.
Vice President
Quality & Analytical Chemistry
Amy Waterhouse
Vice President
Regulatory & Government Affairs
G. Eric Davis
Vice President
General Counsel & Secretary
Mark Wood
Vice President
Human Resources
Steven Jungles
Vice President
Supply Chain
Daniel Maher
Vice President
Product Development
Board of Directors
Jean-Jacques Bienaimé
Chief Executive Officer
BioMarin Pharmaceutical Inc.
Joseph Klein, III
Managing Director
Gauss Capital Advisors, LLC
Pierre Lapalme
Former President &
Chief Executive Officer
North America Ethypharm, Inc.
Michael Grey
President &
Chief Executive Officer
SGX Pharmaceuticals, Inc.
Alan Lewis, Ph.D.
President &
Chief Executive Officer
Novocell, Inc.
Elaine Heron, Ph.D.
Chairman &
Chief Executive Officer
Labcyte Inc.
Randy Meier
Executive Vice President,
Eye Care Business and
Operations, and
Chief Financial Officer
Advanced Medical Optics, Inc.
Forward-Looking Statement: This Annual Report contains ‘forward-looking statements’ as defined under securities laws.
These statements can generally be identified 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 could differ significantly 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 can-
didates, 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 influence 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 reflect later
events or circumstances or to reflect the occurrence of unanticipated events.
Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC. Naglazyme® and BioMarin are registered trademarks of
BioMarin Pharmaceutical Inc.
Jean-Jacques Bienaimé
Chief Executive Officer
Jeffrey Cooper
Senior Vice President,
Chief Financial Officer
Emil Kakkis, M.D., Ph.D.
Senior Vice President,
Chief Medical Officer
Stephen Aselage
Senior Vice President,
Global Commercial Operations
Robert Baffi, Ph.D.
Senior Vice President,
Technical Operations
Stuart Swiedler, M.D., Ph.D.
Senior Vice President,
Clinical Affairs
G. Eric Davis
Vice President,
General Counsel & Secretary
Mark Wood
Vice President,
Human Resources
Phenylase Core Team
Top row, left to right: James Dickow
(Director, Cell Culture), Gia DePillis, Ph.D.
(Director, Regulatory Affairs),
Laurie Tsuruda, Ph.D., D.A.B.T.
(Associate Director, Pharmacology/
Toxicology), Marlyn Morimoto, MS
(Sr. Financial Analyst), Paul Fitzpatrick,
Ph.D. (Principal Scientist, Research and
Development), Mubarack Muthalif, Ph.D.,
MBA (Senior Program Manager, Product
Development/ Phenylase Core Team
Leader) and Julie Wilson, Ph.D.
(Senior Product Manager).
In the Laboratory
Second row, left to right:
Carroll Henschell (Study Monitor,
Pharmacology/Toxicology) and
Sean Bell, Ph.D.
(Scientist, Research & Development).
Kuvan Core Team
Third row, left to right: Mark Henderson,
Ph.D. (Associate Director, Quality
Assurance), Alejandro Dorenbaum. M.D.
(Sr. Medical Director), Benjamin Dewees,
RAC (Senior Manager, Regulatory Affairs),
Julie Schraeder (Director, Finance),
Dong Wei, Ph.D. (Senior Program
Manager, Product Development),
Dan Oppenheimer, Ph.D. (Senior Director,
Program Management / Kuvan Program
Leader), Sandra Shpilberg, MBA (Director
of Marketing), V. Miles Rios, Jr., BA, MA
(Associate Director, Clinical Operations).
Representatives From
European Sales Team
Bottom row, left to right:
Philippe Carteron de Balmont (Country
Manager, Switzerland, France, &
BeNeLux), Guy Eggleton (Director of
Operations, BioMarin Europe Ltd.),
David Boothe, BSc (Director of Marketing,
BioMarin Europe) and Felis Iglesias
(Country Manager, Spain, Portugal).
Less than 10 years after opening our doors, we are approaching the antici-
pated launch of our third product. Our proven ability to deliver products to
market has transformed BioMarin from a product development-focused
company to a fully integrated commercial company with growing product
revenues and an exciting product pipeline. As important as our commercial
success, is the fact that our products are bringing the promise of new thera-
peutics to patients around the world suffering from rare genetic diseases.
Building A Meaningful Revenue Base
The efficiency and speed of our research, development and manufacturing
efforts is at the heart of our ability to quickly bring products to market. We
have successfully advanced each of our two flagship drugs, Aldurazyme®
(galsulfase) and Naglazyme® (laronidase), from research to commercializa-
tion in about five years. Revenues for both products increased substan-
tially in 2006, as we further expanded our reach to patients and secured
regulatory approvals worldwide. This sales growth, coupled with substan-
tial improvements to our capital structure, has significantly improved the
company’s financial profile.
2006 marked important progress in the commercialization efforts of
Aldurazyme. By the end of the year, worldwide sales increased 26 percent
to $96.3 million and as a result, BioMarin’s share of the profit in BioMarin/
Genzyme LLC increased 63.6% over fiscal 2005.
In 2006, Naglazyme, our first independently launched therapy for MPS VI,
was introduced in Europe after launching in the U.S. in mid-2005. When
considered as a stand-alone business, this product became profitable in
its first year on the market, and with $46.5 million in sales, we have clearly
established our ability to independently launch products for rare diseases.
This success now positions BioMarin as a commercial partner of choice for
marketing highly-specialized pharmaceutical products addressing the needs
of patients with rare diseases.
Expanding Product Profile
Growing revenues generated by Aldurazyme and Naglazyme are helping
us advance additional compounds in our product pipeline that target both
orphan genetic diseases and other conditions such as cardiovascular dis-
ease. This will present substantially larger market opportunities to augment
our current enzyme replacement franchise.
KuvanTM (sapropterin dihydrochloride), a small molecule oral therapeutic for
the treatment of phenylketonuria (PKU), has demonstrated strong safety
and efficacy in Phase 3 clinical trials and has been granted Fast Track status
by the FDA. This product, co-developed with Merck Serono, will be the
first approved drug treatment available for PKU, a metabolic disease that
affects more than 50,000 people in the developed world. Pending prior-
ity review designation and a favorable regulatory review, U.S. approval for
Kuvan is expected in late 2007. It will be the third drug we have success-
fully advanced from research to commercialization in less than five years.
The same enzyme cofactor found in Kuvan, 6R-BH4, has also been found
to play a key protective role in the cardiovascular system. To leverage this
asset in markets beyond PKU, we are conducting several proof-of-concept
studies for a variety of cardiovascular indications.
Finally, 2006 marked significant progress in our preclinical development of
PhenylaseTM, an enzyme substitution therapy for the treatment of severe
forms of PKU. We are currently performing additional preclinical studies with
the goal of filing an IND by the end of 2007 and initiating clinical studies in
early 2008.
Looking Forward
We are proud of our track record of bringing innovative, high-value biophar-
maceuticals to market. By leveraging clear-cut development strategy and
efficient, adaptive clinical development models we have achieved regula-
tory approvals far faster than the industry average and the results of our
progress are now becoming more tangible in the form of improved financial
performance. 2006 was a year of growth and progress in our efforts to
provide increased value to both investors and patients.
I would like to thank all of our employees and partners for their hard work
and commitment to excellence. We appreciate your continued support of
the company and look forward to keeping you informed of our progress
throughout the year.
Sincerely,
Jean-Jacques Bienaimé
Chief Executive Officer
2006 Milestones
Commercial Success
BioMarin has transformed from a product
development-focused company to a fully
integrated commercial company with
rapidly growing product revenues and an
exciting product pipeline.
Global Expansion
In 2006, BioMarin further established its
commercial presence in the United States
and Europe and is now expanding into
Latin America.
Improved Financial Profile
2006 was marked by significant increases
in product sales, a decrease in net loss,
the conversion of debt to common stock,
and an increasing cash balance. This has
enabled BioMarin to increase funding of
growth opportunities.
Proven Business Model
After less than four years in develop-
ment, BioMarin’s third product, Kuvan, is
expected to be approved in the U.S. by
the end of 2007, further demonstrating the
company’s ability to recognize unique
opportunities and develop them quickly.
Expanding Market Opportunities
BioMarin is now evaluating 6R-BH4 for
a variety of cardiovascular indications.
If successful, these programs could
significantly expand the company’s global
market potential.
Fulfilling Unmet Needs
BioMarin is bringing the promise of new
therapeutics to thousands of patients
worldwide who might otherwise go
untreated. In less than 10 years, the
company has developed and commer-
cialized two breakthrough drugs for rare
genetic diseases and is well positioned
to address many others with its exciting
product pipeline.
BioMarin Pharmaceutical Inc.
105 Digital Drive
Novato, CA 94949
Tel: 415.506.6700 415.
Fax: 415.382.7889
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BRINGING THEPROMISETOMARKET
CORPORATE INFORMATION
EXPERIENCED MANAGEMENT
A MESSAGE FROM THE CEO
2006 Annual Report
Stock Performance
BioMarin develops and commercializes
innovative biopharmaceuticals for serious
diseases and medical conditions. The
company’s portfolio comprises two
approved products and multiple clinical
and preclinical product candidates.
Corporate Headquarters
Transfer Agent
BioMarin
Nasdaq Stock Market (U.S.)
Nasdaq Biotech Index
The following graph compares the cumulative
total stockholder return with the cumulative
total return of the Nasdaq Stock Market
(U.S.) and the Nasdaq Biotechnology Index,
assuming a $100 investment in BioMarin’s
common stock on December 31, 2002 and
reinvestment of dividends during the period.
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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
Stock Listing
BioMarin Pharmaceutical Inc.
is listed on the Nasdaq
Global Market and the
SWX Swiss Exchange under
the symbol BMRN.
Independent Accountants
KPMG LLP
San Francisco, CA
Mellon Investor Services LLC
480 Washington Boulevard
Jersey City, NJ 07310
Tel: 800-522-6645 (Domestic)
201-680-6578 (International)
Executives
Jean-Jacques Bienaimé
Chief Executive Officer
Jeffrey Cooper
Senior Vice President
Chief Financial Officer
Emil Kakkis, M.D., Ph.D.
Senior Vice President,
Chief Medical Officer
Stephen Aselage
Senior Vice President
Global Commercial Operations
Robert Baffi, Ph.D.
Senior Vice President
Technical Operations
Stuart Swiedler, M.D., Ph.D.
Senior Vice President
Clinical Affairs
Jeff Ajer
Vice President
Sales & Marketing Operations
William Aliski
Vice President &
General Manager
European Operations
R. Andrew Ramelmeier, Ph.D.
Vice President
Manufacturing and
Process Development
Victoria Sluzky, Ph.D.
Vice President
Quality & Analytical Chemistry
Amy Waterhouse
Vice President
Regulatory & Government Affairs
G. Eric Davis
Vice President
General Counsel & Secretary
Mark Wood
Vice President
Human Resources
Steven Jungles
Vice President
Supply Chain
Daniel Maher
Vice President
Product Development
Board of Directors
Jean-Jacques Bienaimé
Chief Executive Officer
BioMarin Pharmaceutical Inc.
Joseph Klein, III
Managing Director
Gauss Capital Advisors, LLC
Pierre Lapalme
Former President &
Chief Executive Officer
North America Ethypharm, Inc.
Michael Grey
President &
Chief Executive Officer
SGX Pharmaceuticals, Inc.
Alan Lewis, Ph.D.
President &
Chief Executive Officer
Novocell, Inc.
Elaine Heron, Ph.D.
Chairman &
Chief Executive Officer
Labcyte Inc.
Randy Meier
Executive Vice President,
Eye Care Business and
Operations, and
Chief Financial Officer
Advanced Medical Optics, Inc.
Forward-Looking Statement: This Annual Report contains ‘forward-looking statements’ as defined under securities laws.
These statements can generally be identified 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 could differ significantly 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 can-
didates, 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 influence 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 reflect later
events or circumstances or to reflect the occurrence of unanticipated events.
Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC. Naglazyme® and BioMarin are registered trademarks of
BioMarin Pharmaceutical Inc.
Jean-Jacques Bienaimé
Chief Executive Officer
Jeffrey Cooper
Senior Vice President,
Chief Financial Officer
Emil Kakkis, M.D., Ph.D.
Senior Vice President,
Chief Medical Officer
Stephen Aselage
Senior Vice President,
Global Commercial Operations
Robert Baffi, Ph.D.
Senior Vice President,
Technical Operations
Stuart Swiedler, M.D., Ph.D.
Senior Vice President,
Clinical Affairs
G. Eric Davis
Vice President,
General Counsel & Secretary
Mark Wood
Vice President,
Human Resources
Phenylase Core Team
Top row, left to right: James Dickow
(Director, Cell Culture), Gia DePillis, Ph.D.
(Director, Regulatory Affairs),
Laurie Tsuruda, Ph.D., D.A.B.T.
(Associate Director, Pharmacology/
Toxicology), Marlyn Morimoto, MS
(Sr. Financial Analyst), Paul Fitzpatrick,
Ph.D. (Principal Scientist, Research and
Development), Mubarack Muthalif, Ph.D.,
MBA (Senior Program Manager, Product
Development/ Phenylase Core Team
Leader) and Julie Wilson, Ph.D.
(Senior Product Manager).
In the Laboratory
Second row, left to right:
Carroll Henschell (Study Monitor,
Pharmacology/Toxicology) and
Sean Bell, Ph.D.
(Scientist, Research & Development).
Kuvan Core Team
Third row, left to right: Mark Henderson,
Ph.D. (Associate Director, Quality
Assurance), Alejandro Dorenbaum. M.D.
(Sr. Medical Director), Benjamin Dewees,
RAC (Senior Manager, Regulatory Affairs),
Julie Schraeder (Director, Finance),
Dong Wei, Ph.D. (Senior Program
Manager, Product Development),
Dan Oppenheimer, Ph.D. (Senior Director,
Program Management / Kuvan Program
Leader), Sandra Shpilberg, MBA (Director
of Marketing), V. Miles Rios, Jr., BA, MA
(Associate Director, Clinical Operations).
Representatives From
European Sales Team
Bottom row, left to right:
Philippe Carteron de Balmont (Country
Manager, Switzerland, France, &
BeNeLux), Guy Eggleton (Director of
Operations, BioMarin Europe Ltd.),
David Boothe, BSc (Director of Marketing,
BioMarin Europe) and Felis Iglesias
(Country Manager, Spain, Portugal).
Less than 10 years after opening our doors, we are approaching the antici-
pated launch of our third product. Our proven ability to deliver products to
market has transformed BioMarin from a product development-focused
company to a fully integrated commercial company with growing product
revenues and an exciting product pipeline. As important as our commercial
success, is the fact that our products are bringing the promise of new thera-
peutics to patients around the world suffering from rare genetic diseases.
Building A Meaningful Revenue Base
The efficiency and speed of our research, development and manufacturing
efforts is at the heart of our ability to quickly bring products to market. We
have successfully advanced each of our two flagship drugs, Aldurazyme®
(galsulfase) and Naglazyme® (laronidase), from research to commercializa-
tion in about five years. Revenues for both products increased substan-
tially in 2006, as we further expanded our reach to patients and secured
regulatory approvals worldwide. This sales growth, coupled with substan-
tial improvements to our capital structure, has significantly improved the
company’s financial profile.
2006 marked important progress in the commercialization efforts of
Aldurazyme. By the end of the year, worldwide sales increased 26 percent
to $96.3 million and as a result, BioMarin’s share of the profit in BioMarin/
Genzyme LLC increased 63.6% over fiscal 2005.
In 2006, Naglazyme, our first independently launched therapy for MPS VI,
was introduced in Europe after launching in the U.S. in mid-2005. When
considered as a stand-alone business, this product became profitable in
its first year on the market, and with $46.5 million in sales, we have clearly
established our ability to independently launch products for rare diseases.
This success now positions BioMarin as a commercial partner of choice for
marketing highly-specialized pharmaceutical products addressing the needs
of patients with rare diseases.
Expanding Product Profile
Growing revenues generated by Aldurazyme and Naglazyme are helping
us advance additional compounds in our product pipeline that target both
orphan genetic diseases and other conditions such as cardiovascular dis-
ease. This will present substantially larger market opportunities to augment
our current enzyme replacement franchise.
KuvanTM (sapropterin dihydrochloride), a small molecule oral therapeutic for
the treatment of phenylketonuria (PKU), has demonstrated strong safety
and efficacy in Phase 3 clinical trials and has been granted Fast Track status
by the FDA. This product, co-developed with Merck Serono, will be the
first approved drug treatment available for PKU, a metabolic disease that
affects more than 50,000 people in the developed world. Pending prior-
ity review designation and a favorable regulatory review, U.S. approval for
Kuvan is expected in late 2007. It will be the third drug we have success-
fully advanced from research to commercialization in less than five years.
The same enzyme cofactor found in Kuvan, 6R-BH4, has also been found
to play a key protective role in the cardiovascular system. To leverage this
asset in markets beyond PKU, we are conducting several proof-of-concept
studies for a variety of cardiovascular indications.
Finally, 2006 marked significant progress in our preclinical development of
PhenylaseTM, an enzyme substitution therapy for the treatment of severe
forms of PKU. We are currently performing additional preclinical studies with
the goal of filing an IND by the end of 2007 and initiating clinical studies in
early 2008.
Looking Forward
We are proud of our track record of bringing innovative, high-value biophar-
maceuticals to market. By leveraging clear-cut development strategy and
efficient, adaptive clinical development models we have achieved regula-
tory approvals far faster than the industry average and the results of our
progress are now becoming more tangible in the form of improved financial
performance. 2006 was a year of growth and progress in our efforts to
provide increased value to both investors and patients.
I would like to thank all of our employees and partners for their hard work
and commitment to excellence. We appreciate your continued support of
the company and look forward to keeping you informed of our progress
throughout the year.
Sincerely,
Jean-Jacques Bienaimé
Chief Executive Officer
2006 Milestones
Commercial Success
BioMarin has transformed from a product
development-focused company to a fully
integrated commercial company with
rapidly growing product revenues and an
exciting product pipeline.
Global Expansion
In 2006, BioMarin further established its
commercial presence in the United States
and Europe and is now expanding into
Latin America.
Improved Financial Profile
2006 was marked by significant increases
in product sales, a decrease in net loss,
the conversion of debt to common stock,
and an increasing cash balance. This has
enabled BioMarin to increase funding of
growth opportunities.
Proven Business Model
After less than four years in develop-
ment, BioMarin’s third product, Kuvan, is
expected to be approved in the U.S. by
the end of 2007, further demonstrating the
company’s ability to recognize unique
opportunities and develop them quickly.
Expanding Market Opportunities
BioMarin is now evaluating 6R-BH4 for
a variety of cardiovascular indications.
If successful, these programs could
significantly expand the company’s global
market potential.
Fulfilling Unmet Needs
BioMarin is bringing the promise of new
therapeutics to thousands of patients
worldwide who might otherwise go
untreated. In less than 10 years, the
company has developed and commer-
cialized two breakthrough drugs for rare
genetic diseases and is well positioned
to address many others with its exciting
product pipeline.
BioMarin Pharmaceutical Inc.
105 Digital Drive
Novato, CA 94949
Tel: 415.506.6700 415.
Fax: 415.382.7889
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