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Having successfully pivoted our product pipeline to include treatments that address larger, genetically driven conditions, we have set the stage for significant growth over the coming years. At our core and consistent with our original mission, we blaze scientific and medical trails to better the lives of those suffering from genetic diseases. Our R&D engine is more productive than ever, and we expect to advance many early-stage candidates in 2022.

Commercial Strength

In 2022, we anticipate increased demand for nearly all our commercial brands. We have expanded our expertise into the commercialization of innovative therapies for genetic conditions with larger patient populations.

Launching a product in Europe ahead of the United States was a first for BioMarin, and our experienced commercial teams are managing the dual launches as planned.

Pipeline Pivot

We have successfully pivoted our pipeline to include genetic diseases with larger patient populations and more common diseases with a genetic component. Our roots in rare disease give us the confidence that few, if any, companies are better positioned to make a big impact on more and more people with genetic disease than BioMarin.

We have a deep R&D pipeline with a targeted focus in key therapeutic areas, including approximately 30 clinical and preclinical candidates.

World Class Manufacturing Capability

With over two decades of operations, BioMarin has become a leader in biologic production from supporting R&D, interacting with regulatory authorities, and supplying demand for our commercial therapies. The breadth and depth of our manufacturing capabilities has generally grown commensurately with our overall growth.

At the forefront of one of the newest advancing fields in biotechnology, we built one of the first gene therapy manufacturing facilities of its kind, spanning 18,000 square feet with the ability to produce up to 10,000 doses per year. From enzymes to gene therapies, our owned manufacturing facilities allow us to maintain high quality standards, along with the efficient production and delivery capability to meet clinical and commercial demands. Our facilities include three cGMP (current Good Manufacturing Practices) manufacturing plants across Novato, California, and Shanbally, Ireland.

Financial Foundation

Our financial foundation is supporting our accelerated growth plan in 2022, as we expect to turn the corner to sustainable profitability this year. This is an important achievement marking the beginning of the next stage of growth for BioMarin. We have built an organization that can support both continued product approvals and innovative pipeline growth, while at the same time generating sustainably increasing profits and positive operating cash flows.

We believe that the strength of our base business, the recent approvals and launches, the potential commercial prospects of our late-stage pipeline, and the increasing number of opportunities in our early-stage pipeline set us up for success well into this decade and beyond.

We also believe that our diversified global business, spanning over 75 countries, will take BioMarin to the next level of growth and transformation. We have invested in the infrastructure to support this growth and look forward to leveraging these capabilities in the coming months and years.

Corporate Citizenship

In addition to our R&D, commercial, and financial strengths, we are also focused on fostering a diverse, equitable, and inclusive workforce. Diversity, equity, and inclusion (DEI) are the sum of many parts, and for our employees to reach their full potential, we must support them in every way—from embracing unique backgrounds and experiences, to applying fair and equitable opportunities for all. For this reason, we take a holistic approach to DEI, constantly examining all areas of our business to ensure they align with our DEI mission. I am proud of how we continue to advance DEI across our company. At the end of last year, our global employee population represented approximately equal numbers of women and men at nearly every level. People of Color represented 45% percent of all U.S. BioMarin employees at the end of 2021. We also focus on achieving full equity in both base pay and total compensation for all U.S. employees and offer a number of learning opportunities available globally that promote an inclusive environment.

As a global corporate citizen, BioMarin commits to operating in a manner that protects the environment and provides a safe and healthy workplace for our personnel and the communities in which we operate. Creating a cleaner, healthier, and more sustainable world is consistent with our values: motivated by our desire to help improve lives, driven by our pioneering spirit, and guided by our passion for science, we have a long history of reducing our impact on the environment. To that end, in 2021, we expanded our greenhouse gas disclosure and laid out a roadmap for further actions in the coming years.

Translating Genetic Discoveries into Transformative Medicines

In closing, while we expect 2022 to be a transitional year in our long-term strategy, we are pleased to see many of BioMarin’s long-time goals start to materialize this year, which include building an enterprise that can support both continued product approvals and innovative pipeline growth, while at the same time generating sustainably increasing profits and positive operating cash flows. The financial, commercial, and regulatory momentum at BioMarin is strong, and we are grateful to all of BioMarin’s stakeholders for the continued support throughout our journey. I especially want to thank BioMarin employees for their continued commitment to developing the essential medicines that help so many.

At BioMarin, our efforts are directed towards transforming lives through genetic discovery. As we celebrate BioMarin’s 25th anniversary, we are well-positioned to deliver on our commitment to our current and future patients as we turn the corner on profitability, allowing us to potentially have an even larger impact on more lives in the future.

Jean-Jacques Bienaimé
Chairman and Chief Executive Officer
BioMarin Pharmaceutical Inc.

– April 12, 2022

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 having set the stage for significant growth over the coming years; BioMarin’s expectation of turning the corner to sustainable profitability this year and of its ability to generate sustainably increasing profits and operating cash flows; BioMarin being set up for success well into this decade and beyond; BioMarin’s expectation that VOXZOGO has the potential to be its largest opportunity to date, with a foundation for continued growth and potential registrations in new markets, and the demand associated with these markets; BioMarin’s expectation that it will advance many early-stage candidates in 2022; BioMarin’s anticipation of increased demand for all of its commercial products with the exception of Kuvan; BioMarin’s expectation that its therapies to treat mucopolysaccharidosis (Types IVA and VI) will contribute significantly to revenue growth this year; BioMarin’s confidence that few, if any, companies are better positioned to make a big impact on more and more people with genetic disease than BioMarin; the continued clinical development and commercialization of BioMarin’s commercial products and product candidates and the timing of such development and commercialization, including, BioMarin’s plan to resubmit its BLA for valoctocogene roxaparvovec to the FDA; the possible approval and commercialization of BioMarin’s product candidates, the expected contributions of these product candidates to top-line growth, if approved; actions by regulatory authorities and the timing of such actions; BioMarin’s belief that its diversified global business will take BioMarin to the next level of growth and transformation; and other clinical development, regulatory interactions, manufacturing and commercial operations in 2022 and beyond. 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 ability to successfully commercialize its current or future commercial products; the results and timing of current and planned preclinical studies and clinical trials, as well as the potential impact of the COVID-19 pandemic on BioMarin’s ability to continue such preclinical studies and clinical trials and the timing of such 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, including the potential impact of the COVID-19 pandemic on the regulatory authorities’ abilities to issue such decisions and the timing of such decisions; 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, 2021, 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.

Our Pipeline

We take on the biggest challenges in rare disease.

Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address.

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