BioMarin Pharmaceutical
Encyclopedia
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.
Biotechnology
Biotechnology is a field of applied biology that involves the use of living organisms and bioprocesses in engineering, technology, medicine and other fields requiring bioproducts. Biotechnology also utilizes these products for manufacturing purpose...
firm based in Novato, California
California
California is a state located on the West Coast of the United States. It is by far the most populous U.S. state, and the third-largest by land area...
. It has offices and facilities in the US, South America, Asia and Europe. BioMarin's core business and research is in enzyme replacement therapies
Enzyme replacement therapy
Enzyme replacement therapy is a medical treatment replacing an enzyme in patients in whom that particular enzyme is deficient or absent. Usually this is done by giving the patient an intravenous infusion containing the enzyme...
(ERTs). BioMarin is the first company to provide therapeutics for mucopolysaccharidosis
Mucopolysaccharidosis
Mucopolysaccharidoses are a group of metabolic disorders caused by the absence or malfunctioning of lysosomal enzymes needed to break down molecules called glycosaminoglycans - long chains of sugar carbohydrates in each of our cells that help build bone, cartilage, tendons, corneas, skin and...
type I (MPS I), by manufacturing laronidase (Aldurazyme, commercialized by Genzyme
Genzyme
Genzyme Corporation is a fully owned subsidiary of Sanofi-Aventis. Before its acquisition, Genzyme was an American biotechnology company based in Cambridge, Massachusetts. In 2010, Genzyme was the world’s third-largest biotechnology company, employing more than 11,000 people around the world...
Corporation). BioMarin is also the first company to provide therapeutics for Phenylketonuria (PKU).
As of 2005, BioMarin commercialized arylsulfatase B
Arylsulfatase B
Arylsulfatase B is an enzyme associated with mucopolysaccharidosis VI.Arylsulfatase B is among a group of arylsulfatase enzymes present in the lysosomes of the liver, pancreas, and kidneys of animals. The purpose of the enzyme is to hydrolyze sulfates in the body. ARSB does this by breaking down...
(Naglazyme) as an enzyme replacement therapy for the treatment of mucopolysaccharidosis
Mucopolysaccharidosis
Mucopolysaccharidoses are a group of metabolic disorders caused by the absence or malfunctioning of lysosomal enzymes needed to break down molecules called glycosaminoglycans - long chains of sugar carbohydrates in each of our cells that help build bone, cartilage, tendons, corneas, skin and...
VI (MPS VI), and in 2007 a drug version of tetrahydrobiopterin
Tetrahydrobiopterin
Tetrahydrobiopterin or sapropterin is a naturally occurring essential cofactor of the three aromatic amino acid hydroxylase enzymes, used in the degradation of amino acid phenylalanine and in the biosynthesis of the neurotransmitters serotonin , melatonin, dopamine, norepinephrine ,...
(Kuvan), the first medication-based intervention to treat phenylketonuria
Phenylketonuria
Phenylketonuria is an autosomal recessive metabolic genetic disorder characterized by a mutation in the gene for the hepatic enzyme phenylalanine hydroxylase , rendering it nonfunctional. This enzyme is necessary to metabolize the amino acid phenylalanine to the amino acid tyrosine...
.
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
PARP inhibitor
PARP inhibitors are a group of pharmacological inhibitors of the enzyme poly ADP ribose polymerase . They are developed for multiple indications; the most important is the treatment of cancer...
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-Diaminopyridine
3,4-Diaminopyridine is an organic compound with the formula C5H3N2. It is formally derived from pyridine by substitution of the 3 and 4 positions with an amino group....
(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
National Health Service
The National Health Service is the shared name of three of the four publicly funded healthcare systems in the United Kingdom. They provide a comprehensive range of health services, the vast majority of which are free at the point of use to residents of the United Kingdom...
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.