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
FDA
After an initial rejection, BioMarin has finally secured the FDA’s approval for Roctavian, the first gene therapy in the U.S. for the most common form of the bleeding disorder.
Regeneron envisions new dosing for Eylea’s label and it’s the moment of truth for BioMarin’s gene therapy for hemophilia A. For that and more, see inside.
BridgeBio posted data Monday from its small but highly anticipated Phase II study of infigratinib in achondroplasia - a drug that could give BioMarin a run for its money.
BioMarin Pharmaceutical announced the FDA no longer plans to hold an advisory committee meeting to review its BLA for Roctavian an AAV gene therapy for adults with severe hemophilia A.
A request for additional data may cause a 3-month delay for potential approval of BioMarin Pharmaceutical’s hemophilia A gene therapy.
During its third-quarter earnings report, BioMarin noted that the FDA planned to hold an advisory committee meeting to discuss the company’s gene therapy for hemophilia A.
BioMarin Pharmaceuticals announced Friday it was shedding around 4% of its global workforce, or around 120 jobs, in an effort to improve its operational efficiency.
Two years after receiving a Complete Response Letter for its hemophilia gene therapy, BioMarin is heading back to the FDA with stronger and more robust data for potential approval.
The Institute for Clinical and Economic Review (ICER) concluded that even at a hefty lifetime cost of $2.5 million, gene therapies for hemophilia A and B are worth it.
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