BioMarin Pharmaceutical Inc. | 2009 Annual Report Racing against time annaBelle : age 3 : MPS iVa Patient “Our greatest hOpe is that a drug will be available in time tO help Our sweet girl.” therapy for MPS IVA which is currently in human clinical trials to test its safety and efficacy. Thus far, preliminary data sug- gest improvements in endurance and pulmonary function. In clinical studies for the com- pany’s similar FDA-approved drugs for MPS I and MPS VI the company observed similar measures of efficacy. “Our greatest hope is that a drug will be available in time to help our sweet girl.” Pa g e 2 | B i o M a r i n P h a r M a c e u t i c a l i n c . | 2 0 0 9 a n n u a l r e P o rt Pa g e 1 | B i o M a r i n P h a r M a c e u t i c a l i n c . | 2 0 0 9 a n n u a l r e P o rt “Every minute of every day counts for us. Because every day that goes by without a treatment, we know that MPS IVA is taking its toll on our daughter’s body. We are literally in a race against time to get ahead of the damage caused by this disease, and as parents, we cannot watch this happen without a fight,” said Stephanie, Annabelle’s mother.Stephanie and her husband Austin have spent the past three years in a desperate chase to find a treatment to save their daughter’s life and alleviate the crippling effects of MPS IVA. This rare genetic disorder, also known as Morquio A Syndrome, causes skeletal dysplasia, spinal malformation, heart and respira-tory failure and other severe or-gan damage. Since her diagno-sis at 6 months, Annabelle has braved countless doctors’ visits, invasive surgery and a lifetime of pain. Others like her have been fighting the disease for years. These patients receive constant medical attention and remedial care for their symptoms, but still await a treatment that will ad-dress the underlying cause and effects of their disease. BioMarin has developed a potential new BarBara K. Burton, M.D. ProfeSSor of PeDiatricS : northweStern uniVerSity feinBerg School of MeDicine Director, PKu anD MetaBolic DiSeaSe clinic : chilDren’S MeMorial hoSPital chicago, il “while extraOrdinary prOgress has been made in recent years, it is crucial that we wOrk tOgether tO further enable drug develOpment fOr rare disOrders.” “Too often by the time patients counselors, nurses, nutrition- “While extraordinary progress has make it to my clinic, irreversible ists, and research and education been made in recent years to damage has already occurred. coordinators provide supportive improve the quality of patients’ Then, once an accurate diagno- medical care to help address lives, it is crucial that we—clini- sis is established, patients and patients’ most immediate needs. cians, researchers, patient advo- their families are faced with the These may include pain relief, cacy groups and pharmaceutical depressing reality that there may finding ways to increase mobility, companies—work together to be no drugs available to treat or addressing their sense of further enable drug development their diseases. So it’s our re- isolation by connecting them for rare disorders. We must con- sponsibility to always try to help with other patients. Specific tinue to provide hope for patients patients in some way now.” needs of patients with rare and make sure they are reaching Geneticist Dr. Barbara Burton has treated patients with rare disorders for 33 years. Daily, she and her team of genetic genetic disorders are complex their full potential.” and vary considerably. Pa g e 2 | B i o M a r i n P h a r M a c e u t i c a l i n c . | 2 0 0 9 a n n u a l r e P o rt Peter l. SaltonStall, PreSiDent anD ceo : Diane DorMan, Vice PreSiDent, PuBlic Policy national organization for rare DiSorDerS [norD] “We have a patient-driven mission A rare “orphan” disease is one “We sometimes think of NORD as at NORD. Our goal is to work with government partners such as the NIH (National Institutes of Health) and FDA, along with patient organizations, academic institutions and biopharmaceu- tical companies like BioMarin to speed up the process of developing safe and effective treatments for people with rare diseases,” said Peter L. Salton- that affects fewer than 200,000 the hub of a wheel because we Americans. According to the have the singular ability to bring ments for rare diseases. Today, NIH there are 7,000 of these dis- people and organizations together the group continues its long eases that affect an estimated in ways that will ultimately benefit legacy of serving the needs of 30 million people in the United patients,” added Diane Dorman, patients with rare diseases. Simi- create financial incentives for companies to develop treat- States alone. Approximately NORD’s Vice President of Public 360 orphan drugs and biologics Policy (pictured below; left). have been approved by the FDA. While estimates indicate that 15 million Americans now benefit from these products, millions NORD, a U.S. based patient ad- vocacy organization, is respon- sible for spearheading orphan drug legislation, specifically the Orphan Drug Act of 1983, to lar sister organizations such as EURORDIS, the European Rare Disease Organization, are also active internationally. stall, NORD President and CEO more remain untreated. (pictured below; right). Pa g e 4 | B i o M a r i n P h a r M a c e u t i c a l i n c . | 2 0 0 9 a n n u a l r e P o rt “Our gOal aT NOrD is TO wOrk wiTh gOverNmeNT parTNers, paTieNTs aND iNDusTry TO speeD up The prOcess Of DevelOpiNg TreaTmeNTs fOr peOple wiTh rare Diseases.” henry J. fuchS, M.D. : executiVe Vice PreSiDent, chief MeDical officer “biOmarin is pOised tO cOntinue tO make significant cOntributiOns tO medicine and tO the lives Of patients with seriOus, unmet medical cOnditiOns.” Pa g e 6 | B i o M a r i n P h a r M a c e u t i c a l i n c . | 2 0 0 9 a n n u a l r e P o rt “I am really proud to work at BioMarin. This company has already helped patients with rare diseases enjoy better lives. And we have a pipeline that is both broad and deep. In the relatively near term, we will be evaluating products in studies designed to support new product approvals, products in mid-stage clinical testing to prove important clinical hypotheses, and new products about to enter the first phase of clinical testing. The team that has already accomplished a lot on behalf of patients is growing, both in number and impact. Their lead-ership will inspire new genera-tions of product development and approval activities. BioMarin has been, is and will continue to be a world class scientific organization. We ask challenging questions about why patients suffer, about how to alleviate that suffering and how to turn ideas into new medicines quickly.”“With explosions in our under-standing of the genetic basis of disease, BioMarin is poised to continue to make significant contributions to medicine and to the lives of patients with serious, unmet medical conditions. My heartfelt thanks go to our investi-gators, patients, families and care-givers, as well as the employees of BioMarin who work tirelessly to help alleviate suffering.” inVeSting in the future : increaSing Manufacturing caPacity fOllOwing the recent cOmpletiOn Of a new expansiOn prOject, biOmarin will mOre than dOuble its clinical and cOmmercial manufacturing capacity thrOughOut the next five years. BioMarin is investing in valuable mercial manufacturing capacity. slated for FDA approval in early infrastructure to keep pace with Kuvan® is produced off site at 2011. The new flexible design will international demand for drugs contract manufacturing facilities accommodate a wide range of to treat rare genetic diseases. in Switzerland. Firdapse™ is pro- production systems to support Currently, the company produc- duced at contract manufacturing future clinical and commercial es two products, Naglazyme® facilities in France. manufacturing needs for Aldura- and Aldurazyme®, at its manu- facturing facilities in Novato, California. Following the recent completion of a new expansion project, the company will more than double its clinical and com- The addition of this new facility will provide a total of 70,000 square feet of manufacturing space at BioMarin, Novato. Commissioned in 2010, it is zyme and Naglazyme and, if approved, GALNS and PEG-PAL. Pa g e 8 | B i o M a r i n P h a r M a c e u t i c a l i n c . | 2 0 0 9 a n n u a l r e P o rt Michael VellarD, Ph.D. : Director anD PrinciPal ScientiSt, reSearch & DeVeloPMent “after spending years wOrking On a drug, it is amazing tO finally witness the first injectiOn and see a patient’s subsequent imprOvements in health.” “I’ve spent my entire career look- irreversible? These diseases are “Developing biologics is a pain- “We have a pioneer spirit at ing for cures for rare genetic debilitating and often progress staking and exacting science, BioMarin and share a keen diseases. It’s been a very per- quickly. There’s simply no time but we have to work quickly and sense of urgency to help sick sonal quest for me because I’ve to spare when you are racing to efficiently, being mindful of the patients. This continually chal- experienced firsthand what it’s save lives,” said Dr. Michael Vel- costs. From a research perspec- lenges and inspires us. After like to live with the uncertainty lard (pictured below). tive, it is often frustrating for us spending years working on the of tomorrow. My young niece, Anne-Claire, has a lysosomal storage disease. Every passing day brings uncertainty: what symptoms will appear next week, and next month? How much more damage will be Dr. Vellard and his team of researchers at BioMarin have developed a promising new compound for the treatment of MPS IVA (Morquio A Syndrome) which is now under evaluation in human clinical trials. to see that while we may have development of a drug, it is developed a viable treatment amazing to finally witness the first for an unmet medical need, this injection, and see a patient’s sub- is only the first step in a lengthy sequent improvements in health.” process to bring the drug directly to patients.” Pa g e 1 0 | B i o M a r i n P h a r M a c e u t i c a l i n c . | 2 0 0 9 a n n u a l r e P o rt BioMarin’S Peg-Pal PrograM core teaM “it takes a lOt Of teamwOrk tO meet aggressive timelines... we’re wOrking On enOrmOusly impOrtant therapies tO help patients whO have nO Other hOpe.” Pa g e 1 2 | B i o M a r i n P h a r M a c e u t i c a l i n c . | 2 0 0 9 a n n u a l r e P o rt “We put a lot of pressure on ourselves to develop drugs quickly. It takes a lot of teamwork to meet aggressive timelines, rigorous product specifications and surmount what seems like endless regulatory hurdles. We’re working on enormously important therapies to help patients who have no other hope. The clock is ticking fast and we have to work together to meet our goals and overcome every single challenge,” said Dr. Mubarack Muthalif, BioMarin’s Associate Director of Product Development (pictured below; seated).Biopharmaceutical product development requires visionary leadership and the close collabo-ration of multiple cross-functional experts. At BioMarin, program development teams work togeth-er to advance individual product candidates from research, into the clinic and on to regulatory approval and commercialization. We strive to do it in record time. The team pictured here is in charge of the navigation and execution of the company’s second PKU program, PEG-PAL, a product candidate that is designed to offer additional ben-efits to patients with the disorder. Another important project also in development is a real-time por-table blood Phe monitor which will allow patients and physicians to better manage the disease. Pictured here are several members of BioMarin’s PEG-PAL core team including (left to right): Paul Fitz-patrick, Ph.D.; Mubarack Muthalif, Ph.D.; Jeri Beltman, Ph.D.; Saba Sile, M.D.; and Mari Maurer. aMy waterhouSe : Vice PreSiDent, regulatory anD goVernMent affairS “we have been very successful in defending the use Of nOvel efficacy assessments and statistical methOds fOr use in Our pivOtal studies that have enabled rapid develOpment times.” “Seeing the profound rates of “Because we are often in the “We operate under very tight milestone, especially when most morbidity and debilitating position of developing the very timelines, and quite frankly, INDs are just three volumes symptoms of rare diseases, it first treatment for an orphan dis- there’s no trick to it. There are in size. And over the past four is abundantly clear that time ease, there is no clear regulatory periods of time when extraordi- years, we’ve obtained market- is of the essence for patients approval pathway in place. We nary effort is required to meet ing approvals for Naglazyme in and their families. We have work very hard to ensure that the our deadlines and it helps to more than 50 countries across worked closely with regulatory regulatory agencies understand know the sacrifices we make Europe, Asia, and North and agencies to streamline process- the diseases and the impact that during these crunch periods are South America. But what’s most es in order to provide therapies our drug has upon patients’ lives. supporting an important cause.” amazing is to witness the un- to improve patient outcomes.” We have been very successful in defending the use of novel effi- cacy assessments and statistical methods for use in our pivotal studies that have enabled rapid development times.” “We thrive on meeting rigorous demands. Filing the 37-volume IND for Kuvan in 2008, for example, was an exhilarating believable real-life stories of pa- tients who experience firsthand the benefits of our therapies.” Pa g e 1 4 | B i o M a r i n P h a r M a c e u t i c a l i n c . | 2 0 0 9 a n n u a l r e P o rt Julie, an industrial interior designer, is also looking forward to future advancements in PKU treat- ment. “I can’t wait until a blood Phe monitoring device is available,” Julie said. “Having access to immediate results will help me quickly respond to my Phe levels, especially while training for sports and planning for pregnancy.” Please see www.bmrn.com for important product and safety information. Julie : age 28 : PKu Patient “advancements in the treatment Of pku have made a huge impact in my life in an incredibly shOrt periOd Of time. i feel a majOr imprOvement, especially when i train fOr runs and triathlOns.” Pa g e 1 6 | B i o M a r i n P h a r M a c e u t i c a l i n c . | 2 0 0 9 a n n u a l r e P o rt “Advancements in the treatment of PKU have made a huge impact in my life in an incredibly short period of time. I’ve had PKU all my life—28 years—and the advances made just within the last five years blow the previous 23 years out of the water!”Julie has a rare genetic disorder, PKU (phenylketonuria), and has been on treatment with Kuvan® (sapropterin dihydrochloride) Tablets since 2005 when she enrolled in BioMarin’s Phase I clinical trials. “Initially, I enrolled in the Kuvan clinical study to help younger people with PKU to have more options,” she said. “I sort of felt it was too late for me. I was 23 then and believed I had pretty good control of my diet and couldn’t do much more than that. But almost immediately I became one of the highest responders in my clinic! Now, five years later, I feel a major improvement, espe-cially when I train for runs and tri-athlons, and my Phe levels remain well within the normal range.”Currently, BioMarin’s second prod-uct candidate for PKU, PEG-PAL is in human clinical trials. The com-pany is also planning to develop an at-home Phe monitoring device, much like a portable glucose moni-tor used by people with diabetes. Jean-JacqueS BienaiMÉ : chief executiVe officer “We’ve got the racing spirit here at BioMarin but it’s not one driver that makes things happen. It’s about a cohesive team work- ing together to accomplish one goal. Meeting that goal is the driving force behind everything we do. That goal is to quickly bring important therapies to patients with rare diseases.” “biOmarin has brOught three prOducts tO market in just 12 years—a track recOrd that far surpasses traditiOnal biOpharmaceutical develOpment timelines.” “Team BioMarin has brought PKU and soon, Lambert Eaton “Quite candidly, we only have a three products to market in Myasthenic Syndrome (LEMS). limited amount of time and we just 12 years—a track record While impressive, we cannot just have to maximize our resources, that far surpasses traditional cruise, satisfied with our past passion and professional exper- biopharmaceutical development successes; we have to think tise in the race to come up with timelines. But what separates proactively and take measured new therapeutic solutions for this company from others is risks. We have to keep moving, patients with rare diseases.” our drive to develop therapies growing and developing, try new for patients who have no other things, seek alternate directions, treatment options. Our therapies move faster and maximize every are the only commercially avail- opportunity to make a difference able pharmaceutical treatments in patients’ lives. We might get a for patients with MPS I, MPS VI, bit bruised up in the process, but when we accomplish all that, it’s an incredibly moving experience.” Pa g e 1 8 | B i o M a r i n P h a r M a c e u t i c a l i n c . | 2 0 0 9 a n n u a l r e P o rt exPanDing gloBal MarKet oPPortunitieS to fulfill unMet MeDical neeDS corPorate inforMation tremendOus BioMarin is leveraging its global efforts have yielded higher than Initiative for Consumers for its commercial infrastructure to expected growth rates and the Facebook PKU Awareness Month prOgress is being meet increasing demands for commercial use of Naglazyme. web page engaging thousands made in bOth its commercial products. The Currently, multiple additional of patients with PKU. Promising company anticipates the launch markets are being assessed. post-marketing data for Kuvan BioMarin PharMaceutical inc. DeVeloPS anD coMMercializeS innoVatiVe BioPharMaceuticalS for SeriouS DiSeaSeS anD MeDical conDitionS. the coMPany’S ProDuct Portfolio coMPriSeS four aPProVeD ProDuctS anD MultiPle clinical anD non-clinical Drug ProDuct canDiDateS. will also extend the benefits of this treatment to many other patients. new and existing markets in the of its fourth product, Firdapse™ (amifampridine phosphate) for Lambert Eaton Myasthenic Syn- drome (LEMS) in Europe early identificatiOn in the second quarter of 2010, Of patients whO can benefit while clinical and regulatory teams coordinate with the FDA to establish development plans for approval and commercializa- frOm naglazyme tion in the United States. therapy. Since the launch of Naglazyme in 2005, tremendous progress in both new and existing markets is being made in the identification of patients who can benefit from Naglazyme therapy. Aggressive international sales and marketing The graphic shown here illus- trates BioMarin’s direct, partnered and named-patient distribution efforts for Nagla- zyme. In total, Naglazyme is treating hundreds of patients in multiple countries around the world. Sales of Aldurazyme also continue with steady growth. Kuvan commercialization efforts in the U.S. incorporate a variety of innovative approaches to engage clinicians and patients. BioMarin received the 2009 Medical Marketing and Media Award for Best Interactive 27 24 2 15 68 BioMarin Direct Presence DistriBution Partner in Place naMeD Patient sales – no DistriButor Markets Being assesseD total countries Pa g e 2 0 | B i o M a r i n P h a r M a c e u t i c a l i n c . | 2 0 0 9 a n n u a l r e P o rt Gordon Vehar, Ph. D. Vice President, Research Ed Von Pervieux Vice President & Chief Information Officer Jackie Walling, M.D., Ph.D. Vice President, Clinical Development Amy Waterhouse Vice President, Regulatory & Government Affairs Board of directors Jean-Jacques Bienaimé Chief Executive Officer Michael Grey Former President & Chief Executive Officer, Auspex Pharmaceuticals, Inc. Elaine Heron Chairman & Chief Executive Officer, Amplyx Pharmaceuticals, Inc. Pierre Lapalme Former President & Chief Executive Officer, North America Ethypharm, Inc. V. Bryan Lawlis President and Chief Executive Officer, Itero Biopharmaceuticals, Inc. Alan Lewis President & Chief Executive Officer, Juvenile Diabetes Research Foundation Randy Meier Executive Vice President & Chief Financial Officer, Teleflex, Inc. corporate Headquarters BioMarin Pharmaceutical Inc. 105 Digital Drive Novato, CA 94949 Tel. 415.506.6700 Fax. 415.382.7889 Email. ir@bmrn.com www.bmrn.com stock Listing BioMarin Pharmaceutical Inc. is listed on the Nasdaq Global Select Market under the symbol BMRN (NASDAQ: BMRN). independent accountants KPMG LLP San Francisco, CA transfer agent Bank of New York Mellon Transfer Agent Bank of New York Mellon 480 Washington Boulevard Jersey City, NJ 07310 U.S. Tel. 800.522.6645 International Tel. 201.680.6578 executives Jean-Jacques Bienaimé Chief Executive Officer Stephen Aselage Executive Vice President and Chief Business Officer Henry J. Fuchs, M.D. Executive Vice President, Chief Medical Officer Robert A. Baffi, Ph.D. Executive Vice President, Technical Operations Jeffrey H. Cooper Senior Vice President, Chief Financial Officer G. Eric Davis Senior Vice President and General Counsel Daniel P. Maher Senior Vice President, Product Development Mark Wood Vice President, Human Resources Jeff Ajer Vice President, Sales & Marketing Operations Luisa Bigornia Vice President, Intellectual Property Lewis Chapman Vice President, Global Marketing Joshua A. Grass Vice President, Corporate & Business Development Steven Jungles Vice President,Supply Chain James K. Lennertz, Jr Vice President, Commercial Operations Brian Mueller Vice President, Controller Charles A. O’Neill, Ph.D. Vice President, Pharmacological Sciences Dan Oppenheimer, Ph.D. Vice President, Portfolio Strategy Leonard Post, Ph.D. Vice President, Drug Discovery R. Andrew Ramelmeier Vice President, Manufacturing & Process Development Victoria Sluzky, Ph.D. Vice President, Quality & Analytical Chemistry Eduardo Thompson Vice President, General Manager, Latin America FORWARD-LOOkING STATEMENT: This Annual Report contains ‘forward-looking statements’ as defined under securities laws. These statements can generally be iden- tified by the use of terminology such as ‘believes’, ‘expects’, ‘anticipates’, ‘plans’, ‘intends’, ‘may’, ‘will’, ‘projects’, ‘continues’, ‘estimates’, ‘potential’, ‘opportunity’, and so on. The company’s actual results or experience may differ 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 candidates, its ability to successfully market products and other factors discussed in the enclosed Form 10-k and the section entitled ‘Risk Factors’ therein. One should not place undue 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 under- take 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. BioMarin®, Naglazyme® and kUVAN® are registered trademarks of BioMarin Pharmaceutical Inc. Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC. FirdapseTM is a trademark of BioMarin Huxley Ltd. Please see www.bmrn.com for important product and safety information. © 2010. BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical inc. 105 Digital Drive novato, ca 94949 tel: 415.506.6700 fax: 415.382.7889 investor relations: ir@bmrn.com www.bmrn.com B i o M a r i n P h a r M a c e u t i c a l i n c . | l e T T e r T o o u r s h a r e h o l d e r s The year 2009 will be remembered by many as one of The mosT TurbulenT economic Times in recenT hisTory. aT biomarin, however, we had The disTincT forTune To noT only weaTher The volaTile economic sTorms, buT To Thrive. increased global demand for our Therapies and aggressive pipeline expansion will be The hallmarks of 2009. The year was not without its existing markets exceeded our discussions with the FDA to challenges, but by leveraging expectations with a 27 percent establish development plans the confidence and security that increase in net product sales for Firdapse in the U.S. and we comes with having strong cash over 2008, and third party sales plan to submit an IND for the reserves, a solid financial footing of Aldurazyme showed modest PARP inhibitor BMN-673 at the and an unwavering focus on build- gains as the number of patients end of 2010. ing long term value, we further on therapy continued to increase. solidified our position in the rare Total annual net revenues for Ku- disease arena. Throughout the van were $76.8 million, compared coming year, we will continue to to $46.7 million in 2008, and year enhance our product pipeline and end cash, cash equivalents and maximize investment opportuni- short and long term investments ties to fuel long term growth. posted at $470.5 million for the Highlights of the year include strong sales of our three commer- cialized products, the acquisition of Huxley Pharmaceuticals and several promising compounds, the advancement of several year. Another important achieve- ment was the completion of construction of our new manufac- turing facility in Novato, which will more than double our manufac- turing capacity. clinical and nonclinical programs, Encouraging early data from the and the anticipated launch of our GALNS Phase I/II program for fourth and newly acquired prod- MPS IVA leads us to anticipate a uct, FirdapseTM (amifampridine pivotal Phase III trial by the end phosphate) for LEMS in Europe. of 2010. As for PEG-PAL, the The company’s total net product Phase II study is progressing and revenues increased 25 percent we expect to report some results over 2008, posting at $315.7 mil- in the third quarter of 2010. We lion. Naglazyme sales in new and continue to build important data on Kuvan therapy outcomes and we have initiated an exciting new program to develop a real time blood Phe monitoring device that will enable patients to better track and manage their PKU. Our BMN- 195 small molecule upregulator of utrophin for Duchenne Muscular Dystrophy has advanced from preclinical status to Phase I clini- cal trials. Soon we will begin We are pleased with our perfor- mance in 2009 and well posi- tioned to meet our goals for 2010 as we look forward to an exciting year ahead. We are focused on carefully managing our pipeline and commercial products with the goal of continuing double-digit revenue growth in the coming years and to maximize long term value to BioMarin, patients and our shareholders. I thank all of our employees for their expertise, passion and hard work throughout the year. I would also like to thank patients and physicians for partnering with us to develop important thera- peutics for rare diseases. I look forward to keeping you informed and sharing future developments with you as 2010 progresses. Thank you for your support. Sincerely, Jean-Jacques Bienaimé Chief Executive Officer B i o M a r i n P h a r M a c e u t i c a l i n c . | 2 0 0 9 y e a r i n r e v i ew biomarin pharmaceuTical develops, manufacTures and commercializes innovaTive biopharmaceuTicals for serious diseases and medical condiTions. The company’s producT porTfolio comprises four approved Therapies and mulTiple clinical and nonclinical producT candidaTes. 2009 milesTones Investment In the Future soon be launched in Europe and BioMarin recently completed con- struction of its expanded manufac- turing facility in Novato, California. This new site will accommodate a wide range of production systems the company is currently explor- ing development strategies for the U.S. market, along with additional indications for future development. to support future clinical and com- PeoPle Power mercial manufacturing needs for BioMarin continues to increase Aldurazyme and Naglazyme and, recruiting efforts to support the if approved, GALNS and PEG-PAL. company’s rapid growth. At the InternatIonal CommerCIal Growth close of 2009 the employee roster contained a total of 714 people. In 2010, with even more attention Sales of BioMarin’s three com- focused on pipeline development mercial products continued and further global expansion, the to increase in 2009, driven by company has plans to increase higher than expected international personnel by up to 15 percent in demand for Naglazyme. The com- the next nine months. pany’s fourth and newly acquired product, Firdapse™ for LEMS, will Biomarin PiPeline 5-yr revenue growth nonClInICal testInG Phase 1 Phase 2 Phase 3 Bla : nDa : maa CommerCIalIZeD Naglazyme® for MPS VI Aldurazyme® for MPS I Kuvan® for PKU Firdapse™ for LEMS BMN-110 GALNS for MPS IVA PEG-PAL for PKU BMN-195 Utrophin upregulator for Duchenne Muscular Dystrophy BMN-103 α-glucosidase (GAA) for Pompe Disease BMN-168 6R-BH4 Prodrug BMN-185 IgA Protease for IgA Nephropathy BMN-673 PARP Inhibitor for Genetically Defined Cancers BMN-111 350 300 250 200 150 100 50 0 ) s n o i l l i m n i $ ( e u n e v e r l a t o t 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 may 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 candidates, its ability to successfully market products and other factors discussed in the enclosed Form 10-K and the section entitled ‘Risk Factors’ therein. One should not place undue 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. BioMarin®, Naglazyme® and KUVAN® are registered trademarks of BioMarin Pharmaceutical Inc. Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC. FirdapseTM is a trademark of BioMarin Huxley Ltd. Please see www.bmrn.com for important product and safety information. © 2010. Biomarin Pharmaceutical inc. 5 0 0 2 6 0 0 2 7 0 0 2 8 0 0 2 9 0 0 2 Financial StrengthRobust product sales fueled growth in 2009 and the company expects to be cash flow positive in 2010. To maximize long term value to patients and sharehold-ers, management is focused on carefully managing the advancing pipeline portfolio with the goal to continue double-digit revenue growth in the coming years. PiPeline exPanSionBioMarin continues to make significant investments in research and development to ensure continued growth and innovation. The current pipeline includes a collection of promising clinical and nonclinical programs.