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
Gene therapy has been a dream for many researchers for decades—inserting a healthier gene into patients’ cells to replace defective genes and curing diseases. One disease that would seem to be a good target for gene therapy is hemophilia.
It’s hard to believe 2018 is half over. There have been plenty of interesting and important stories in the biopharma space this year. Here’s a look at our top 10 stories so far, counting down from 10 to the number one story.
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.
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.
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