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

BioMarin is a world leader in developing therapies to treat rare genetic diseases, and after almost two decades, our extensive research and development efforts continue to expand the boundaries of medicine. Guided by our commitment to the rare disease community, we seek to rapidly develop first-in-class or best-in-class therapies that will make a meaningful difference to small patient populations.

We have five commercialized products and multiple clinical and pre-clinical product candidates in our pipeline. Our clinical success on behalf of our rare disease patients has produced robust revenue growth from our existing products. We expect to turn the corner toward profitability by achieving non-GAAP breakeven or better in 2017 and bottom line growth in the years to follow. In 2015, strong demand and revenue growth across our commercialized products delivered total revenue of $889.9 million, representing an 18.8% year over year increase that was driven by the continued strong global launch of Vimizim®.

In 2015, we laid the groundwork to submit up to two new marketing applications by year end that could result in two potential global product launches in 2017. We expect to submit marketing applications in the U.S. and Europe by mid-2016 for cerliponase alfa to treat a form of Batten disease, which is a rapidly progressing, fatal neurodegenerative disease with no approved treatments. The majority of affected children lose their ability to walk and talk by approximately six years of age and usually die between the ages of ten and 16 years of age. We have developed a potential first enzyme replacement therapy administered directly into the brain ventricles for a form of Batten disease, and it could ultimately change the course of this terrible disease.

Earlier this year, BioMarin also announced positive Phase 3 data for pegvaliase, an investigational drug to treat adults with phenylketonuria (PKU) who have inadequately controlled blood phenylalanine (Phe) levels. This was the first placebo controlled study that showed improvement in Phe levels in PKU patients who did not have to follow a Phe-restricted diet, which does not allow any protein. We intend to submit a marketing application for pegvaliase by the end of the year subject to further discussions with the FDA.

In 2015, our business development efforts resulted in two key strategic transactions to refocus our pipeline, reduce some future R&D expenses and to increase our near-term revenues. The first was the sale of an oncology therapy in development, talazoparib, and the second was the acquisition from Merck Serono of all rights outside of the U.S. to Kuvan® and pegvaliase, with the exception of Kuvan in Japan.  We are now selling Kuvan in approximately 60 countries and expect an additional $70-80 million in revenue attributable to Kuvan in 2016.

In 2015, we completed the acquisition of Prosensa and added Duchenne muscular dystrophy products to our portfolio. We also submitted marketing applications for Kyndrisa in the U.S. and in Europe for a potential treatment for boys with exon 51 skipping amenable Duchenne muscular dystrophy. Based on unfavorable opinions from health authorities on Kyndrisa, in May 2016 we made the difficult decision to discontinue our current experimental drugs for Duchenne. We now plan to invest in research of next generation oligonucleotides with the goal of making a safe and effective treatment available for boys with this devastating disorder.

BioMarin has a vital portfolio of products from early to mid-stage of development, including vosoritide, an experimental treatment for achondroplasia, a form of dwarfism. In 2015, we announced Phase 2 data with vosoritide for children with achondroplasia and are encouraged by the evidence of activity. Based on the observed changes in growth, this therapy could potentially allow children with achondroplasia to resume a normalized growth rate. By developing a therapy that addresses the root cause of achondroplasia, we also hope to address the associated complications, such as disproportionate bone growth. We have begun discussions with health authorities on a path forward for registration enabling studies.

We are also excited about an early phase trial with BMN 270, our gene therapy product for patients with hemophilia A. If successful, gene therapy could 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, who have conducted hundreds of pre-clinical studies and are building on decades of experience in gene therapy construction.

Finally, 2015 marked the tenth anniversary of the approval of Naglazyme® in the U.S. Naglazyme created the commercial foundation for BioMarin to develop and successfully launch our other approved products, including Vimizim, our most successful launch to date. Engaging with the MPS patient community for more than a decade has been one of the most fulfilling aspects of BioMarin’s work. We have three approved therapies for different types of MPS and have a fourth in development.

In addition to maintaining our deep relationships with patient communities, our success hinges on building and maintaining long-term relationships with physicians, academic researchers, our employees, elected officials, regulatory bodies, investors and the local communities where we operate. Inspired by patients with rare diseases and their families, we are driven to identify exciting scientific discoveries in monogenetic diseases and translate those discoveries into commercially viable products that make a meaningful difference in patients’ lives.

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.

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




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



Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361

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