| Type | Public company |
|---|---|
| Traded as | NASDAQ: BMRN |
| Industry | Biotechnology |
| Founded | 1997 |
| Headquarters | Novato, CA, USA |
| Key people | Jean-Jacques Bienaime |
| Revenue | US$376M (FY 2010)[1] |
| Operating income | US$1.67M (FY 2010)[1] |
| Net income | US$206M (FY 2010)[1] |
| Total assets | US$1.26B (FY 2010)[2] |
| Total equity | US$717M (FY 2010)[2] |
| Employees | 700[3] |
| Website | www.biomarinpharm.com |
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) 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.[4]
In 2009, BioMarin acquired Huxley Pharmaceuticals, Inc. (Huxley), which had rights to a proprietary form of 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate[5]. 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[6].
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[7]. 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[8]. 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)[9].
In 2010, BioMarin became involved in controversy [10][11] 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.[12]