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
Horizon Pharma and the National Organization for Rare Disorders (NORD) launched a new campaign today that recognizes innovation in rare disease therapeutics since the passing of the Orphan Drug Act in 1983.
FDA
The U.S. Food and Drug Administration (FDA) gave BioMarin Pharmaceutical a thumbs-up for its Palynziq (pegvaliase-pqpz) to treat phenylketonuria (PKU).
Most investors thought 2018 would be a big year for mergers and acquisitions in the biopharma industry because of changes to the tax law, and so far they’ve been right.
In order to drive the manufacture of affordable generic drugs, the U.S. Food and Drug Administration is naming names of companies that have attempted to block competition.
As we get closer to seeing the U.S. Food and Drug Administration green light the first gene therapy for hemophilia, analysts estimate that the cost of the probable one-time treatment could have a whopping price-tag of $1.5 million.
After nearly a year without a permanent chief executive officer Advaxis, Inc. has a new hand on the wheel. This morning the company announced Kenneth A. Berlin will take over the helm of the company as president and CEO effective immediately.
Here are five possible acquisition targets to keep an eye on.
This year wasn’t big for mergers and acquisitions in biopharma, particularly compared to 2015 and 2016.
A look at the FDA’s long-awaited guidelines on how it will regulate regenerative medicine products, including stem cells.
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