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 2018, BioMarin continued to pursue bold science and to work toward changing the practice of medicine in rare genetic diseases. We drove our business forward as we accomplished one product approval in the U.S. and reached several key milestones for two product candidates in Phase 3 of development. Scientifically, we seek to correct serious genetic diseases either by replacing what is missing or prompting the body itself to start providing what it needs. Our goal is to help the communities of people with rare diseases perform biologically the same as or similarly to people without disease.

Unmatched Commitment to PKU Community

In May 2018, BioMarin received approval from the U.S. Food and Drug Administration (FDA) for Palynziq® (pegvaliase-pqpz) Injection, the first enzyme substitution therapy for phenylketonuria (PKU). This therapy significantly reduces blood phenylalanine (Phe) concentrations in adult PKU patients through a completely new mechanism of action. Palynziq is bringing many patients to within their target Phe ranges for the first time, and in some cases reaching normal or near-normal levels of Phe. The U.S. launch of Palynziq began last July and continues to progress well. In March 2019, we announced that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), adopted a positive opinion for the company’s Marketing Authorization Application (MAA) for Palynziq. We expect the European Commission, which typically adheres to the recommendation of the CHMP, to make its final decision on our market authorization for Palynziq in the second quarter of 2019.

Throughout the biopharmaceutical industry, BioMarin’s long-term commitment to PKU patients and their families is unmatched. Over the last 15 years, BioMarin has conducted 49 clinical trials in PKU, published 54 articles in scientific journals and supported 52 publications developed externally to further enhance our understanding of the disease. In addition to having two approved products to treat PKU, Kuvan® (sapropterin dihydrochloride) and Palynziq, we are on track to submit a clinical trial application with regulatory authorities in the second half of 2019 for BMN 307, a gene therapy treatment for PKU.

Gene Therapy Leadership

We are proud of our leadership in gene therapy with valoctocogene roxaparvovec for severe hemophilia A, which is already in Phase 3 of development, and we believe well ahead of other gene therapies for severe hemophilia A. Gene therapy represents a technology that has the potential not only to dramatically improve patient outcomes and the practice of medicine, but to fundamentally shift the economics of healthcare. We have completed enrollment of participants in the Phase 3 study with the 6e13 vg/kg dose that we believe will satisfy accelerated filing requirements with the FDA. We expect to announce prior to the end of 2019 as to whether we will submit a Biologics License Application through an accelerated approval pathway. Concurrently, we expect to complete enrollment of all 130 participants in the full Phase 3 study in the third quarter of this year. And finally, we expect to provide a three-year data update from our ongoing Phase 2 study with the 6e13 vg/kg dose and a two-year update of the 4e13 vg/kg dose by midyear.

Recognizing a need for world class manufacturing capabilities in gene therapy, we invested early and moved quickly to build our own gene therapy facility. As a result, we have more control over the timing and quality of manufacturing than if we had relied on third-party manufacturers. Our facility is capable of producing 4,000 doses of valoctocogene roxaparvovec annually, and with certain upgrades, we believe we can increase that amount to 5,000. The plant is already producing both valoctocogene roxaparvovec and BMN 307 at the scale required for commercial manufacture.

Our development of gene therapies for hemophilia A and PKU is just the beginning of BioMarin’s applications of gene therapies to treat rare diseases. Our team of experts include both the pioneers of gene therapy and the newer talent in the field, who have shown early success in this field. Together, they are driving incredible innovation. You can expect to hear more from us on plans to develop gene therapies in other disease areas.

Potential First Treatment for the Most Common Cause of Disproportionate Short Stature

We are also excited about the potential impact of vosoritide, which is in Phase 3 of development. Vosoritide is an analog of C-type Natriuretic Peptide (CNP) and is being evaluated in children with achondroplasia, the most common form of disproportionate short stature in humans. The next milestone for vosoritide will be the top-line Phase 3 data read-out expected in the fourth quarter of this year. At R&D Day last November, we shared 42-month data that demonstrated cumulative height gain of 5.7 centimeters – a significant outcome for the children in our Phase 2 study. To support marketing applications in the U.S. and Europe, the dataset will consist of the results from the Phase 3 study, the Phase 2 study, which will include 4-5 years of follow up, and natural history data. Furthermore, we are conducting an infant/toddler study in children 0-5 years old that has received substantial interest from families. The scientific and medical experts from an FDA advisory committee stated there is a strong belief that starting treatment for achondroplasia as soon as possible should result in the most dramatic benefit for children with this condition. This currently enrolling study will provide important insight into the potential benefits and risks of starting treatment with vosoritide as early as possible.

Scientific Pioneers

Looking back at BioMarin’s history, Feb. 14, 2019 marked the fifth anniversary of the approval of Vimizim® (elosulfase alfa) for Mucopolysaccharidosis IV Type A or Morquio A. Vimizim is a perfect example of BioMarin’s pioneering spirit. Before Vimizim went into clinical development, a key expert in skeletal dysplasia had expressed concern that an ERT would not be able to penetrate the cells in the bone where the disease did the most damage. We recognized the multi-systemic nature of Morquio A, which gave us the confidence to challenge conventional wisdom and move ahead into the clinic. We were able to show improved outcomes in pulmonary function and endurance. We know we made the right decision because five years after the approval of Vimizim, there are more than 1,300 Morquio A patients on treatment in 52 countries. It’s no wonder that Vimizim is not only our fastest growing product, but was also our largest product by revenues in 2018.

Since the approval of Vimizim, BioMarin obtained regulatory approval for three products in five years, and each one was first-in-class and overcame risks and doubts using robust data and patient experience. With Brineura® (cerliponase alfa), we trusted the science and validated that our approach to treat CLN2, a pediatric neurological lysosomal storage disorder, with an injection of an ERT directly into a child’s brain. This had never been done before in a clinical trial. With Palynziq, we found ourselves yet again in uncharted waters to bring the first enzyme substitution therapy to adult PKU patients. Because it was a non-human enzyme, we had to figure out how to manage the body’s immune response, which the experts felt would be extremely challenging. As pioneers, we do not have the luxury of following a well-trodden path. Instead, we strive to use our scientific knowledge to make wise decisions in the absence of well established medical norms.

As we note our successes from the past year and anticipate continued success in the coming year, we recognize the many people and organizations that have contributed to BioMarin’s achievements: patients and families with rare genetic diseases, patient advocacy organizations, BioMarin employees, physicians, academic researchers, regulatory authorities, government officials, payers and the local communities where we operate. We have the fortitude to embrace risk, pursue bold science and defy conventional wisdom to change the world for patients with rare genetic diseases.


Jean-Jacques Bienaimé
Chairman and Chief Executive Officer
BioMarin Pharmaceutical Inc.
April 25, 2019

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



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Fax: 888.863.3361

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