BioMarin Pharmaceutical Inc.

Our Purpose

Since our founding in 1997, we have applied our scientific expertise in understanding the underlying causes of genetic conditions to create transformative medicines, using a number of treatment modalities.

Using our unparalleled expertise in genetics and molecular biology, we have developed eight important medicines for patients with significant unmet medical need. Our approved therapies treat achondroplasia, severe hemophilia A and several rare inherited and lysosomal storage diseases.

As we have looked to expand our impact, our approach to cutting-edge science has remained the same. We enlist the best of the best – people with the right technical expertise and a relentless drive to solve real problems – and create an environment that empowers our teams to pursue bold, innovative science. Our work is deeply rooted in genetic insights, meaning we target the underlying genetic changes or proximal molecular mechanisms of disease.

With this distinctive approach to drug discovery, we’ve produced a diverse pipeline of commercial, clinical and preclinical candidates that have well-understood biology and provide an opportunity to be first-to-market or offer a substantial benefit over existing therapeutic options.

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770 Lindaro Street
San Rafael, CA 94901

105 Digital Drive
Novato, CA 94949
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NEWS
BioMarin Pharmaceuticals on Monday said it is restricting sales of its hemophilia A gene therapy to three countries in an effort to reduce costs and help the treatment become profitable by 2025.
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The annual observance highlights how far the industry has come and serves as a wake-up call for how far it must still go to meet the needs of patients with rare diseases.
The U.S. Department of Justice has slapped BioMarin Pharmaceutical with a subpoena in connection with its sponsored testing programs for the rare disease therapies Vivizim and Naglazyme.
Activist investor Elliott Investment Management has struck a deal with BioMarin and will add three people to its board of directors with a new stake in the company.
Activist hedge fund investor Elliott Investment Management has bought over a $1 billion stake in BioMarin as the biotech grapples with a leadership change and disappointing sales of its hemophilia A gene therapy.
FDA
The label for the achondroplasia drug, which promotes endochondral bone growth, now covers children under five years of age with the rare genetic disease causing the most common form of dwarfism.
The first half of this year saw milestone approvals for rare disease therapies, and the FDA has several more such decisions on its calendar in the second half of 2023.
FDA
The FDA’s CBER approved nine novel therapies in the first half of 2023, with several more high-profile decisions on deck for the second half of the year.
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