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
The first half of this year saw milestone approvals for rare disease therapies, and the FDA has several more such decisions on its calendar in the second half of 2023.
FDA
The FDA’s CBER approved nine novel therapies in the first half of 2023, with several more high-profile decisions on deck for the second half of the year.
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.
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