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’s Roctavian was granted conditional authorization in the European Union, making it the first gene therapy approved for severe hemophilia A. Now, the company is shooting for U.S. approval.
Sanofi announced positive data for its therapeutic, fitusiran, for treating patients with hemophilia A and B, as well as efanesoctocog alfa therapy for treating hemophilia A.
It was a busy, busy week for clinical trial news and updates, largely driven by the annual ASCO meeting taking place June 3-7 in Chicago.
The U.S. FDA has requested some additional data from BioMarin Pharmaceuticals for the BLA it filed for the hemophilia A therapy valoctocogene roxaparvovec.
The Q1 results for Voxzogo gave BioMarin the confidence to raise its 2022 full-year guidance to between $100 million to $125 million (net product revenue) for the drug.
In honor of World Hemophilia Day, BioSpace takes a look at some of the facts relating to the disease, and some of the highlights in recent hemophilia research and development.
It was a busy week for clinical trial announcements. Here’s a look.
BioMarin Pharmaceuticals has published positive results in the New England Journal of Medicine from its Phase III clinical trial of valoctocogene roxaparvovec.
Silence Therapeutics, Totus Medicines and Poseida Therapeutics all adjust as they welcome new members to their leadership teams.
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