|Traded as||NASDAQ: BMRN|
|Headquarters||Novato, CA, USA|
|Key people||Jean-Jacques Bienaime|
|Revenue||US$376M (FY 2010)|
|Operating income||US$1.67M (FY 2010)|
|Net income||US$206M (FY 2010)|
|Total assets||US$1.26B (FY 2010)|
|Total equity||US$717M (FY 2010)|
BioMarin Pharmaceutical Inc. is a biotechnology firm based in Novato, California. It has offices and facilities in the US, South America, Asia and Europe. BioMarin's core business and research is in enzyme replacement therapies (ERTs). BioMarin is the first company to provide therapeutics for mucopolysaccharidosis type I (MPS I), by manufacturing laronidase (Aldurazyme, commercialized by Genzyme Corporation). BioMarin is also the first company to provide therapeutics for Phenylketonuria (PKU).
As of 2005, BioMarin commercialized arylsulfatase B (Naglazyme) as an enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI), and in 2007 a drug version of tetrahydrobiopterin (Kuvan), the first medication-based intervention to treat phenylketonuria.
In 2009, BioMarin acquired Huxley Pharmaceuticals, Inc. (Huxley), which had rights to a proprietary form of 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate. In 2010, BioMarin was granted marketing approval by the European Commission for 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate for the treatment of the rare autoimmune disease Lambert-Eaton myasthenic syndrome (LEMS). BioMarin launched the product under the name Firdapse.
In 2010, BioMarin acquired LEAD Therapeutics, Inc. (LEAD), a small private drug discovery and early stage development company with key compound LT-673, an orally available poly (ADP-ribose) polymerase (PARP) inhibitor studied for the treatment of patients with rare, genetically defined cancers. This acquisition was followed by the purchase of ZyStor Therapeutics, Inc. (ZyStor), a privately held biotechnology company developing ERTs for the treatment of lysosomal storage disorders and its lead product candidate, ZC-701, a fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for Pompe disease. At its R&D day in October 2010, BioMarin also announced a new program for a peptide therapeutic for the treatment of achondroplasia (BMN-111).
In 2010, BioMarin became involved in controversy surrounding 3,4-Diaminopyridine (3,4-DAP). BioMarin markets a phosphate salt of 3,4-DAP under the name Firdapse. In 2010, BioMarin was granted exclusive licensing rights to Firdapse for 10 years. As a result, the price of a prescribed National Health Service treatment course has increased from £1250 for the unlicensed drug to £44,000 for Firdapse. The company states that prior to its licensing, there was no guaranteed quality control of the product and no way of formally monitoring for uncommon side effects through the regulatory process.
- BioMarin Pharmaceutical (BMRN) annual SEC income statement filing via Wikinvest.
- BioMarin Pharmaceutical (BMRN) annual SEC balance sheet filing via Wikinvest.
- Quackenbush, Jeff (2011-02-14). "BioMarin Expands as It Tests New Drugs". Retrieved 2011-02-14.
- Pollack, A (2007-12-14). "Agency Approves Drug to Treat Genetic Disorder That Can Lead to Retardation". The New York Times. Retrieved 2008-04-03.
- Stevens, Loralee (2009-10-27). "BioMarin Acquires Huxley Pharmaceuticals". North Bay Business Journal. Retrieved 2011-02-14.
- "BioMarin launches LEMS drug Firdapse in European Union; and Genmab gets conditional approval for Arzerra". The Pharma Letter. 2010-04-20. Retrieved 2011-02-14.
- Brown, Steven (2010-02-04). "BioMarin to buy Lead Therapeutics for $18M". San Francisco Business Times. Retrieved 2011-02-14.
- Leuty, Ron (2010-08-17). "BioMarin to buy ZyStor for up to $115M". San Francisco Business Times. Retrieved 2011-02-14.
- Andersen, Karen (2010-10-21). "BioMarin Highlights Diverse Early-Stage Pipeline at R&D Day". Morningstar. Retrieved 2011-02-14.
- Goldberg, Adrian (2010-11-21). "Drug firms accused of exploiting loophole for profit". BBC News. Retrieved 2010-11-21.
- Martin, Daniel (2010-09-27). "Hospitals are forced to use unlicensed medicines to save millions". Daily Mail (London). Retrieved 2010-11-21.
- What makes an orphan drug?. British Medical Journal. 2010-11-10. Retrieved 2010-11-21.