BioMarin Pharmaceutical Inc.
ABOUT
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with multiple commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
770 Lindaro Street
San Rafael, CA 94901
105 Digital Drive
Novato, CA 94949
San Rafael, CA 94901
105 Digital Drive
Novato, CA 94949
NEWS
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.
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.
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.
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.
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.
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