BioMarin Pharmaceutical Inc.
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About BioMarin Pharmaceutical Inc.
At BioMarin, we focus on developing first-in-class and best-in-class therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases. BioMarin remains steadfast to its original mission—to bring new treatments to market that will make a big impact on small patient populations. These patient populations are mostly children, suffering from diseases so rare, that the entire patient population can number as few as 1,000 people worldwide. These conditions are often inherited, difficult to diagnose, progressively debilitating, have few, if any, treatment options, and are usually ignored.
BioMarin offers a friendly, fast-paced, collegial environment where success is measured by both the individual and collective efforts of hundreds of talented scientists, business professionals, technical specialists and experts in a variety of fields from sales and marketing, to finance, to manufacturing, to clinical and regulatory operations.
BioMarin is growing rapidly to support the development and global commercialization of breakthrough drugs to treat rare genetic diseases. The company’s product portfolio comprises six approved products and multiple clinical and pre-clinical product candidates.
You can make it happen at BioMarin, where the unique combination of big-company success in a small-company environment provides unparalleled opportunity for individual contributions and growth.
554 articles with BioMarin Pharmaceutical Inc.
Express Scripts is already known for its role of bringing prescriptions to the doors of consumers. Now the company is moving into a new area of providing medicines—expensive gene therapies.
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.
Total Revenues of $372.8 million in the Quarter
BioMarin to Host Second Quarter 2018 Financial Results Conference Call and Webcast on Thursday, August 2 at 4:30pm ET
BioMarin Pharmaceutical Inc. announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Thursday, August 2, at 4:30 p.m. ET
First-of-Its-Kind Musical to Help Empower the Bleeding Disorder Community to Find and Use their Voices through the Power of Music
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 thi...
BioMarin Doses First Participant in Phase 2 Study of Vosoritide for Treatment of Infants and Young Children with Achondroplasia
Vosoritide represents an innovative therapy to treat the underlying cause of achondroplasia.
BioMarin Pharmaceutical Inc. announced that the Company received $20 million in milestone payments from Pfizer Inc.
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.
Imara Inc. today announced additions to its clinical development team with the appointments of Joe Datt, M.D., as Senior Vice President, Clinical Development, and Siddharth Parulkar as Head, Global Clinical Operations.
The U.S. Food and Drug Administration (FDA) gave BioMarin Pharmaceutical a thumbs-up for its Palynziq (pegvaliase-pqpz) to treat phenylketonuria (PKU).
BioMarin Provides 2 Years of Clinical Data in 6e13 vg/kg Dose from Ongoing Phase 1/2 Study in Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A at World Federation of Hemophilia 2018 World Congress
Eliminated Need for Prophylaxis and No Spontaneous Bleeds in Year 2 Quality of Life Scores Continue to Increase in Year 2
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.
coreHEM Publishes Core Outcome Set for Hemophilia Clinical Trials; Prepares to Launch Next Phase of Work
The coreHEM initiative has announced the publication of a core outcome set for clinical trials of gene therapy in hemophilia.
5/18/2018In 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.
BioMarin Announces First Patient Dosed in Phase 1/2 Study Evaluating Valoctocogene Roxaparvovec Gene Therapy in Severe Hemophilia A Patients with Pre-existing AAV5 Antibodies
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that it has dosed the first patient in a Phase 1/2 study (BMN 270-203) evaluating its investigational gene therapy, valoctocogene roxaparvovec, in severe hemophilia A patients with pre-existing AAV5 antibodies.
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.
- Bank of America Merrill Lynch Health Care Conference on May 15 in Las Vegas