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Welcome to BioMarin's Online Press Room.
Here, we’ve brought together a variety of information in one location to better assist you in your efforts to research and develop news articles. Should you require additional information, please call or e-mail us any time.
Sincerely,
Susan Berg
Corporate Communications
sberg@bmrn.com
Tel. (415) 506-6594
Corporate Overview
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) develops and commercializes promising therapeutics for patients with severe and/or life-threatening diseases. Since it was founded in 1997, the company has successfully advanced three breakthrough products from bench, to market, to patients—a remarkable accomplishment in the biopharmaceutical industry. By cultivating expertise in research, development and manufacturing, and by streamlining clinical and regulatory development, BioMarin is providing rapid access to treatment and support services to patients around the world suffering from rare genetic diseases. Headquartered in Novato, California, the company operates subsidiary offices in the United Kingdom, Europe, Latin America and the Middle East.
BioMarin Products: Delivering Breakthrough Treatments Worldwide
Naglazyme® (galsulfase) is BioMarin’s first independently developed and commercialized product and the first and only enzyme replacement therapy (ERT) for the treatment of MPS VI (mucopolysaccharidosis VI, or Maroteaux-Lamy Syndrome), which is estimated to occur once in every 340,000 births. MPS VI is a progressive lysosomal storage disorder that can cause severe disabilities such as impaired cardiac and pulmonary function, delayed physical development, skeletal and joint deformities, reduced endurance, and impaired vision and hearing. Naglazyme provides patients with a recombinant human version of the enzyme they are lacking. Naglazyme has been shown to improve walking and stair-climbing capacity.
Aldurazyme® (laronidase) is the first and only enzyme replacement therapy for the treatment of MPS I (mucopolysaccharidosis I, also known as Hurler, Hurler-Scheie and Scheie Syndromes), a rare, inherited metabolic disease that is estimated to occur 1:100,000 births. Like Naglazyme, this drug provides individuals with the recombinant version of the enzyme they are lacking. Aldurazyme is indicated for MPS I patients with Hurler, Hurler-Scheie, and Scheie (with moderate to severe symptoms) forms. Aldurazyme has been shown to improve pulmonary function and walking capacity. It has not been evaluated for effects on the central nervous system manifestations of the disorder. Aldurazyme is manufactured by BioMarin and marketed by Genzyme Corporation.
Kuvan® (sapropterin dihydrochloride) Tablets is the first FDA-approved medicine for the treatment of phenylketonuria (PKU), a lifelong genetic metabolic disorder. When left untreated, PKU can result in mental retardation and other neurological problems. Individuals with PKU are born with a deficiency in the activity of the enzyme phenylalanine hydroxylase (PAH) and cannot naturally metabolize phenylalanine (Phe), which is toxic to the brain. Phe is found in most foods including meat, eggs, dairy, pasta, fruits and vegetables. Kuvan is to be used with a Phe-restricted diet. PKU is the most common inherited metabolic disorder, estimated to occur in 1:10,000-15,000 births.
For important safety information, including a boxed warning for Aldurazyme, please visit www.naglazyme.com, www.aldurazyme.com, and/or www.kuvan.com. BioMarin is a multinational commercial biopharmaceutical company. By leveraging its expertise in manufacturing, research, regulatory affairs and global commercial development, the company is making significant strides in delivering first-to-market therapeutics to patients with rare genetic diseases.
Expanding Product Pipeline
BioMarin is working to further diversify its growing product portfolio with various research and development programs, which include an exciting range of breakthrough drug candidates, such as 6R-BH4 for pulmonary hypertension, PEG-PAL (PEGylated recombinant phenylalanine ammonia lysase) for PKU, and GALNS for MPS IVA syndrome. A variety of other promising preclinical programs are also being pursued with the goal of filing one IND per year to ensure continued growth.
Accelerating Commercial Success
BioMarin continues to solidify its commercial presence in the United States and Europe, and is rapidly expanding into other regions such as Latin America and the Middle East. Active efforts are also in progress to maximize the company’s intellectual property position and significantly extend product exclusivity beyond orphan protection timelines in the U.S. and abroad.
