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
Miracles, it seems, have a high price tag. At least, if those miracles are miracle drugs. There’s no doubt that trends in gene therapy and immuno-oncology are producing drugs that are as close to miraculous as we’re likely to get, doing a great job, generally, in beating back diseases that to this point were untreatable or didn’t respond well to other therapies.
BioMarin Pharmaceutical received $20 million in milestone payments from Pfizer after the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) approved its applications for talazoparib.
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.
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