BioMarin Pharmaceutical

BioMarin Pharmaceutical Inc.

Type	Public company
Traded as	NASDAQ: BMRN
Industry	Biotechnology
Founded	1997
Headquarters	San Rafael, CA, USA
Key people	Jean-Jacques Bienaime, Chief Executive Officer Henry J. Fuchs, Chief Medical Officer Dan Spiegelman, Chief Financial Officer
Products	Kuvan, Naglazyme, Aldurazyme, Firdapse, Vimizim
Revenue	US$ 751 million (2014)[1]
Operating income	US$ -133 million (2014)[1]
Total assets	US$ 2.49 billion (2014)[1]
Total equity	US$ 1.52 billion (2014)[1]
Number of employees	1681 (2015)[2]
Website	www.bmrn.com
Footnotes / references [3][4]

BioMarin Pharmaceutical Inc. is a biotechnology company based in San Rafael, California. It has offices and facilities in the United States, South America, Asia, and Europe. BioMarin develops and commercializes innovative biopharmaceuticals for serious, life-threatening rare diseases and medical conditions. 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).

History

BioMarin was founded in 1997 by Christopher Starr Ph.D. and Grant W. Denison Jr. with an investment of a $1.5 million from Glyko Biomedical and went public in 1999.^[5][6]

Business development

In 2002, BioMarin acquired Glyko Biomedical.^[7]

In 2009, BioMarin acquired Huxley Pharmaceuticals, Inc. (Huxley), which had rights to a proprietary form of 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate.^[8] 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.^[9]

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.^[10] 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.^[11] 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).^[12]

In 2012, BioMarin acquired Zacharon Pharmaceuticals, a private biotechnology company based in San Diego focused on developing small molecules targeting pathways of glycan metabolism.^[13]

In 2014, BioMarin acquired a histone deacetylase inhibitor chemical library from Repligen for $2 million with the intention of advancing work toward therapies for Friedreich's ataxia and other neurological disorders.^[14] In November, the comapny announced its intention to acquire Prosensa for up to $840 million.^[15]

Products

As of 2014, BioMarin has five products on the market, each of which is an orphan drug.^[16]

• Tetrahydrobiopterin (branded as Kuvan) (sapropterin dihydrochloride), a small molecule drug for phenylketonuria, introduced in 2007 as the first medication-based intervention to treat phenylketonuria^[17]
• Arylsulfatase B (branded as Naglazyme) (galsulfase), a recombinant protein therapeutic for Maroteaux–Lamy syndrome (also called mucopolysaccharidosis type VI)
• Iduronidase (branded as Aldurazyme), a recombinant protein therapeutic for mucopolysaccharidosis I
• Amifampridine (branded as Firdapse), a small molecule drug for Lambert–Eaton myasthenic syndrome (as of 2013 approved in the EU only)
• Elosulfase alfa (branded as Vimizim), is the only enzyme replacement therapy to address the cause of Morquio A Syndrome (MPS IVA), which affects an estimated 3,000 patients in the developed world. The disease occurs as a result of a deficiency of activity in an enzyme involved in glycosaminoglycan (GAG) metabolism.

Controversies

In 2010, BioMarin became involved in controversy^[18][19] 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 $1,987 for the unlicensed drug to $69,970 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.^[20]

In 2013, BioMarin Pharmaceuticals was at the center of a high profile debate regarding expanded access of cancer patients to experimental drugs.^[21] On the advice of her doctor, Andrea Sloan, a patient with advanced ovarian cancer, requested that the company provide her with access to BMN 673, an unapproved PARP inhibitor drug candidate that had exhibited promising activity in a small Phase 1 clinical trial. The company declined, citing safety concerns.^[22] Ms. Sloan eventually received a similar drug candidate from a different company.^[23]

References

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