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
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.
Biomarin indicated that the FDA had requested more data from non-clinical studies to evaluate the theoretical cancer risk to human trial participants. This is likely to take several quarters.
Novartis CEO Vas Narasimhan and Roche CEO Severin Schwan saw revenue growth for the past year, and each took home more than $12 million in total compensation for 2021.
Heading into the middle of January, companies announced plenty of new clinical trial news. Here’s a look.
The results over the two-year evaluation period for its Phase III GENEr8-1 trial showed consistent clinical benefit from using valoctocogene roxaparvovec.
From the continuing fight against COVID-19 to new companies emerging in exciting therapeutic areas to the people who mattered most, here’s a look at just some of the biggest successes, most dramatic flops – and a few that fall somewhere in between.
FDA
Going through the most newsworthy stories of the year, BioSpace found trends more than one big story, topics that just kept rising again and again. Here’s a look.
FDA
BioMarin Pharmaceutical scored a first on Friday and provided an option for patients with a rare disease for which there is little recourse.
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