Innovative therapeutics

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A Message from the Chairman and CEO

Welcome! Thank you for visiting the BioMarin Pharmaceutical website.

Jean-Jacques Bienaimé - BioMarin Chairman and Chief Executive Officer

In 2017, BioMarin is marking its 20th anniversary. For the last two decades, we have been at the forefront of the development of treatments for rare genetic diseases that mostly affect children. During that time, we brought five products to market, and we are gearing up for a potential sixth. In the past 10 years, BioMarin has experienced tremendous growth, as demonstrated by a more than 1,200% increase in revenues. As we look at what we have accomplished and what lies ahead, we believe that our next 20 years will be even brighter.

Our success as a company is in no small part due to our ability to
focus on the unmet needs of our patients when developing therapies.
We excel at charting new courses to bring a therapy from the lab
bench to the patient’s bedside when none existed previously. Keenly
aware that first-in-class and best-in-class therapies have the potential to
fundamentally change the course of disease, we consistently set a high
bar for ourselves in the drug development process.

For BioMarin, 2016 was a momentous year. We had a number
of important development and regulatory successes. We reported
statistically significant results from each of our clinical studies with
our investigational therapies: pegvaliase for phenylketonuria (PKU),
Brineura for a form of Batten disease, vosoritide for a form of
dwarfism, and proof-of-concept results with BMN 270, a potential
one-time gene therapy treatment for hemophilia A. In addition, our
regulatory submissions for Brineura were accepted in both the United
States and Europe, we reported exciting preliminary results on our
BMN 270 gene therapy program, and we began our Phase 3 study
with vosoritide.

In 2016, total revenues topped $1 billion for the first time, coming
in at $1.12 billion, which represented a 26% increase over 2015.
These results were driven by the approximately 50% year-over-
year combined growth of Vimizim® (elosulfase alfa) and Kuvan®
(sapropterin dihydrochloride). We are also laying the groundwork
for potentially substantial returns going forward. Top line growth in
2017 is expected to be around 15% while expenses are expected to
increase by only about 4%. We expect to achieve this growth in part
by increasing our ex-North America Kuvan revenues and by continuing
to add patients for Vimizim.

We were not able to bring Kyndrisa, an investigational medicine
for exon 51 skip amenable Duchenne muscular dystrophy, to market
in the United States or Europe. We are continuing our early research
and development efforts with the hope of developing more effective
treatments for Duchenne. We also made the difficult decision to
discontinue our BMN 701 (reveglucosidase alfa) development program
for Pompe disease as part of our regular evaluation of our pipeline.
Nevertheless, we managed the business to be prepared for a variety
of circumstances and believe we are in a strong position for the year

Looking forward to 2017, double digit revenue growth is expected to
continue, with total revenue expected to be between $1.25 billion and
$1.30 billion. We also anticipate positive non-GAAP results in 2017.
In the years to come, we intend to steadily increase bottom line results
while continuing to reinvest in our pipeline.

On the development and regulatory side, in April 2017 we received
a positive opinion from the Committee for Human Medicinal Products
of the European Medicines Agency for our Marketing Authorization
Application for Brineura, and we look forward to potential approvals
and launches of Brineura in both the United States and Europe.
In 2017, we also anticipate the pegvaliase filing with the U.S.
Food and Drug Administration expected in the second quarter,
the start of the registration enabling Phase 2b study for BMN 270
as well as additional data from the current Phase 1/2 study, and
the announcement of our next new investigational drug (IND)
candidate. Our new gene therapy manufacturing facility is expected
to be complete by mid-year, enabling us to move the BMN 270
program forward without potential third-party constraints on clinical or
commercial product supply.

BioMarin has a track record of tackling diseases that most other drug
companies won’t or can’t. We know how much every patient with a
rare disease matters and how hard it is for a patient to get diagnosed,
let alone find a treatment. We work as fast as we can because we
know that many patients are waiting, and we may be the only ones
developing a therapy that could help them.

Looking to the future, we are pioneering a gene therapy treatment for
hemophilia A that could, if successful, provide a one-time treatment
for patients. Gene therapy would represent not only a medical first
for hemophilia A patients, but a new technology platform that could
change the way we think about treating rare genetic diseases. We
have assembled a world-class team of scientists and clinicians who
are building on decades of experience in gene therapy and are
bringing our technical operations capabilities to bear to facilitate
Good Manufacturing Practices production in our own facility.

BioMarin creates innovative therapies, which can provide significant
value to our patients and to the health care system. The value of this
innovation is recognized globally, where cost-conscious single-payer
health care systems dominate. The majority of our revenue comes from
outside of the United States where single-payer systems from more than
65 countries currently agree to pay essentially the same price for our
therapies that we currently charge in the United States.

As we celebrate BioMarin’s 20th anniversary, we are proud of what
we have accomplished and continue to build on our success. We
are in the midst of a decade-long transformation from a small, one
or two product company into a profitable, growing company with a
robust portfolio of products, a few of which have significant potential.
We begin 2017 from a position of strength. Our approved products
are expected to drive record revenues, again. We are waiting for
the anticipated approval of Brineura. We intend to file the biologics
licensing application for pegvaliase. We are looking forward to
expanding our pipeline with a new drug candidate when we file an
IND application in the second half of this year.

We thank you for your continued support of BioMarin, and we look
forward to sharing news of our future accomplishments.


Jean-Jacques Bienaimé
Chairman and Chief Executive Officer
BioMarin Pharmaceutical Inc.
April 25, 2017

BioMarin provides breakthrough treatments to patients around the world suffering from rare genetic diseases.




Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU, LEMS



Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361

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