Rare Opportunities, Personal Impact
BioMarin is a world leader in developing and commercializing innovative biopharmaceuticals for rare diseases driven by genetic causes
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.
Time is critical to patients with rare diseases, and we strive to quickly develop important therapies for them. The efficiency and speed of our research, development, manufacturing, and commercial efforts is at the heart of our ability to urgently deliver therapies. Our track record of developing and commercializing new treatments has been significantly faster than the industry average and is ingrained in our culture. CenterWatch, a leading source for global clinical trial information, has named it one of the fastest drug developers in the industry. BioMarin was also ranked tenth on Forbes list of innovative companies in 2015.
As a biopharmaceutical company, our success stems from our focus on science and the hope our therapies can bring to patients. We are world leaders in metabolic disease innovation with multiple products commercialized and a growing pipeline of product candidates to address unmet medical needs. We continue to fuel our R&D engine by looking for opportunities that align with our strengths and competencies. And we relentlessly pursue exciting, early-stage science that has the potential to change the course of disease.
A constant in BioMarin’s culture is how patients continue to drive us. The passion and dedication that our employees bring to work each day is a testament to the inspiration our patients provide, and the knowledge of the impact we can make in their lives.
BIOMARIN AT A GLANCE
|Headquarters:||San Rafael, California|
|Chairman and CEO:
|Revenue 2015:||$890 Million|
|Net Income (Loss) 2015:||($171.8 million)|
|Focus:||Rare and ultra-rare genetic diseases|
|Products:||Vimizim® (elosulfase alfa) for (MPS IVA)
Kuvan® (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution for PKU
Naglazyme® (galsulfase) for MPS VI
Firdapse® (amifampridine phosphate) (currently approved in the EU only) for LEMS
Aldurazyme® (laronidase) for MPS I
|Clinical Pipeline:||Pegvaliase (Peg-Pal): for PKU
Reveglucosidase Alfa (BMN 701): GILT GAA for Pompe Disease
Vosoritide (BMN 111) Analog of CNP: for Achondroplasia
Cerliponase Alfa: for CLN2 disease
BMN 270: AAV-Factor VIII Vector for Hemophilia A
BMN 250: GILT rhNAGLU for Sanfilippo Syndrome/MPS IIIB
Drisapersen: for Duchenne Muscular Dystrophy (exon 51)
BMN 044: for Duchenne Muscular Dystrophy (exon 44)
BMN 045: for Duchenne Muscular Dystrophy (exon 45)
BMN 053: for Duchenne Muscular Dystrophy (exon 53)
BioMarin operates a cGMP (current Good Manufacturing Practices) manufacturing facility located in Novato, California. The facility includes dedicated cGMP process manufacturing, quality control, and research and development laboratory space. In 2011, the company announced that it had acquired a biologics manufacturing plant from Pfizer in Shanbally, Cork, Ireland, which is planned to produce biologics in 2017. In addition to the cGMP facility, BioMarin continues to operate process development and pilot scale laboratories to further evaluate investigational product candidates.
Sales and Marketing
As a multinational commercial biopharmaceutical company, BioMarin provides important therapeutic treatments to thousands of patients worldwide who might otherwise go untreated. Our global salesforce continues to solidify the company’s commercial presence in the United States and Europe, and is rapidly expanding into other regions such as Latin America, the Middle East, and Asia-Pacific.