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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

2018 is already off to a great start at BioMarin. Our success as a company hinges on our ability to develop treatments for rare genetic diseases that target specific molecular alterations that can normalize the biology of a disease. We strive to identify critical markers of the treatment that can reflect a dramatic impact on the patient’s quality of life and survival. We do this by taking advantage of our deep knowledge of genetics, biology, medical science and manufacturing biologics. Our research and development efforts build on our scientific expertise and seek to embrace whatever technology precisely addresses the mechanism of action of a disease.

By demonstrating a dramatic impact on a patient’s disease, we can often compress development times and deliver a treatment to patients in significantly less time than the industry average. Targeting normalization of the biology of the disease is a substantial medical achievement and can provide a strong value proposition for cost-conscious payers around the world.

We are looking forward to reaching several important milestones in our pipeline this year, and we continue to invest in and scale our operations for substantial growth over the coming years.

We achieved our most recent milestone in late March of 2018, when the European Medicines Agency (EMA) accepted our submission of a Marketing Authorization Application for pegvaliase for the treatment of adults with phenylketonuria (PKU). The U.S. Food and Drug Administration (FDA) granted priority review status in August 2017, with an updated Prescription Drug User Fee Act Action Goal Date of May 25, 2018. If approved, pegvaliase would represent our second treatment for PKU and could provide an important treatment option for those adult patients with inadequate blood phenylalanine control on existing management. With more than a decade of experience marketing Kuvan® (sapropterin dihydrochloride) for PKU, BioMarin has created a solid foundation upon which to launch pegvaliase and to build a strong PKU franchise.

Looking back at 2017, we celebrated the first two decades of BioMarin’s history. It was an extraordinary year driven by significant financial, regulatory and clinical achievements.

In 2017, we received FDA and EMA approvals of Brineura® (cerliponase alfa) for the treatment of CLN2, a form of Batten disease, in less than four years from first patient enrolled. Brineura also was a notable scientific accomplishment and achieved many firsts, including the first approved treatment for this form of Batten disease and the first enzyme replacement therapy (ERT) specifically approved for administration directly to the brain. Brineura has received external recognition from scientific, patient and professional organizations. We’re building on our knowledge of delivering an ERT directly to the brain with tralesinidase alfa (BMN 250) for MPS IIIB, an experimental therapy for Sanfilippo B syndrome. Tralesinidase alfa represents a potential fourth BioMarin treatment for a form of mucopolysaccharidosis and our second ERT delivered directly to the brain.

We have also been pioneering a totally new way to treat hemophilia A using gene therapy, a potential one-time treatment. We’re excited to be at the forefront of this transformative technology, and look forward to the possibility of providing a treatment that potentially could normalize Factor VIII levels resulting in physiologic restoration of bleeding control in patients with severe hemophilia A. We continue to explore other potential gene therapy treatments where the biology supports the application of this new modality.

For valoctocogene roxaparvovec specifically, we have seen encouraging safety and efficacy data from our Phase 1/2 study for the treatment of severe Hemophilia A, which resulted in the initiation of our Phase 3 program consisting of two separate Phase 3 studies. One study already began at the end of 2017. Valoctocogene roxaparvovec received PRIME designation from the EMA and Breakthrough designation from the FDA.

In December last year, the New England Journal of Medicine (NEJM) published a peer-reviewed article, which included several authors from BioMarin, on the ongoing Phase 1/2 study to evaluate safety and efficacy of valoctocogene roxaparvovec at the 6e13 vg/kg dose in men with severe hemophilia A. The NEJM article concluded that valoctocogene roxaparvovec is associated with the “sustained normalization” of Factor VIII activity over a period of one year in six of seven study participants who received the 6e13 vg/kg dose with stabilization of hemostasis and a “profound” reduction in Factor VIII use in all seven participants. The article also concluded that safety findings were limited to elevations in liver function tests and notes the relatively small sample size.

In addition, we are seeing enthusiasm from the patient community for a potential life changing therapy delivered via a single IV infusion, which could be an appealing option for those severe patients who deal with frequent joint bleeds and the need to always have access to rescue medications even with regular infusions of their existing therapy.

Further, BioMarin completed one of the first gene therapy manufacturing facilities of its kind in the world, establishing its expertise and leadership in this area. This award-winning facility will produce material for the clinical studies of valoctocogene roxaparvovec and, if approved, for commercial demand.

Our other later-stage development program, vosoritide for achondroplasia, progressed as expected over the year. In the fall of last year, we shared data on vosoritide demonstrating consistent durability and safety over 30 months in our Phase 1/2 study. The global Phase 3 program continues to enroll as planned, and we anticipate full enrollment by mid-year.

In summary, 2017 was not only our 20th anniversary year, but also one of our most productive to date. In 2018, we expect to see continued double-digit revenue growth, and in the years to come we intend to steadily increase bottom line results while continuing to reinvest in our pipeline. Our accomplishments in 2017 have laid the groundwork for even greater productivity and execution in 2018 in clinical development, regulatory interactions, manufacturing and commercial operations.

We are grateful for the support we receive from patients with rare genetic diseases and their families, patient advocacy organizations, BioMarin employees, physicians, academic researchers, regulatory authorities, elected officials, payers and the local communities where we operate. Together, we are able to make a big difference in small patient populations.


Jean-Jacques Bienaimé
Chairman and Chief Executive Officer
BioMarin Pharmaceutical Inc.
August 10, 2018

Forward-Looking Statements

This letter contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about BioMarin’s continued revenue growth; BioMarin’s ability to reach expected milestones for products and product candidates in its pipeline; the continued clinical development and commercialization of BioMarin’s commercial products and product candidates; the possible approval and commercialization of BioMarin’s product candidates; the adequacy of production of valoctocogene roxaparvovec in the Company’s commercial gene therapy manufacturing facility; actions by regulatory authorities; and other clinical development, regulatory interactions, manufacturing and commercial operations in 2018. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin’s success in the commercialization of its current or future commercial products; the results and timing of current and planned preclinical studies and clinical trials; BioMarin’s ability to successfully manufacture its commercial products and product candidates; the content and timing of decisions by the FDA, the European Commission and other regulatory authorities concerning BioMarin’s products and product candidates; the market for BioMarin’s products and product candidates; actual sales of BioMarin’s commercial products; and those factors detailed in BioMarin’s filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption “Risk Factors” in BioMarin’s Annual Report on Form 10-K for the year ended December 31, 2017, as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin 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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