BioMarin Pharmaceutical Inc.

ABOUT

BioMarin is a leading rare disease biotechnology company focused on genetically defined conditions.

Guided by our purpose to develop medicines that make a profound impact on people’s lives, our global teams have delivered a portfolio of therapies since our founding in 1997. Our revolutionary treatments for conditions like achondroplasia (the most common form of dwarfism), PKU (phenylketonuria), CLN2, a form of Batten disease, and a number of forms of MPS (mucopolysaccharidosis) offer new possibilities for patients and families who previously had few, if any, available options. More recently, with the close of the Amicus acquisition, our portfolio has expanded to include therapies for Fabry disease and Pompe disease, expanding our ability to reach more people living with rare genetic conditions.

Our success comes from our unwavering commitment to excellence, our deep understanding of patient needs, our scientific expertise, and our world-class manufacturing capabilities. At the heart of BioMarin is a dedicated team of the brightest minds in the industry working together to deliver innovative therapies to patients and families around the world.

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770 Lindaro Street
San Rafael, CA 94901

105 Digital Drive
Novato, CA 94949
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NEWS
Last week, BioMarin revealed changes to its C-suite; now, the company has announced its second round of layoffs this year, following the termination of 170 employees in May.
As the biotech implements a more focused strategy for its Roctavian hemophilia A gene therapy, BioMarin has recruited two seasoned pharma executives to bolster its C-suite.
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.
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