BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that AnGes MG, Inc. (AnGes), BioMarin's marketing and distribution partner in Japan, has submitted a Biologics License Application (BLA) for Naglazyme(R) (galsulfase) to the Japanese Ministry of Health, Labour and Welfare.
"We are pleased to be working with AnGes to bring the first drug treatment option to MPS VI patients in Japan," said Stephen Aselage, Senior Vice President of Global Commercial Development at BioMarin. "Patient advocacy groups and medical societies in Japan have shown a strong interest in Naglazyme, and we are honored to bring this life-altering therapy to MPS VI patients in the U.S., Europe, and now to other parts of the world."
BioMarin established a marketing and distribution agreement with AnGes in December 2006, through which AnGes obtained exclusive rights to market Naglazyme in the Japanese market. Naglazyme was approved by the U.S. Food and Drug Administration (FDA) in May 2005 and by the European Commission (EC) in January 2006. As the first drug approved for MPS VI, the FDA and EC have both designated Naglazyme as an orphan drug, conferring seven years of market exclusivity in the United States and 10 years of market exclusivity in the European Union. In addition, Naglazyme obtained orphan designation in June 2007 from the Ministry of Health, Labour and Welfare (MHLW) in Japan.
About MPS VI
MPS VI (also known as Maroteaux-Lamy syndrome) is a debilitating, life-threatening genetic disease caused by a deficiency of the enzyme N-acetylgalactosamine 4-sulfatase. This enzyme deficiency leads to the accumulation of certain complex carbohydrates, glycosaminoglycans (GAGs), in the lysosomes, giving rise to progressive cellular, tissue and organ system dysfunction. The majority of individuals with MPS VI die from disease-related complications between childhood and early adulthood. Additional information can be found at http://www.mpsvi.com/.
Naglazyme is the first and only enzyme replacement therapy indicated for the treatment of MPS VI. As the first drug approved for MPS VI, regulatory agencies in both the United States and European Union have granted Naglazyme orphan drug status, which confers seven years and 10 years of market exclusivity, respectively. Additional information can be found at http://www.naglazyme.com/.
Naglazyme is indicated for patients with MPS VI. Naglazyme has been shown to improve walking and stair-climbing capacity.
The most common adverse events observed in clinical trials in Naglazyme-treated patients were headache, fever, arthralgia, vomiting, upper respiratory infections, abdominal pain, diarrhea, ear pain, cough, and otitis media. Severe reactions included angioneurotic edema, hypotension, dyspnea, bronchospasm, respiratory distress, apnea, and urticaria. The most common symptoms of infusion reactions included fever, chills/rigors, headache, rash, and mild to moderate urticaria. Nausea, vomiting, elevated blood pressure, retrosternal pain, abdominal pain, malaise, and joint pain were also reported. No patients discontinued Naglazyme infusions for adverse events and all patients that completed the double-blind portion of the trial continue to receive weekly infusions of Naglazyme. Nearly all patients developed antibodies as a result of treatment, but the level of the immune response did not correlate with the severity of adverse events or impact the improvements experienced in endurance. Because antihistamine use may increase the risk of apneic episodes, evaluation of airway patency should be considered prior to the initiation of treatment. Consideration to delay Naglazyme infusion should be given when treating patients who present with an acute febrile or respiratory illness.
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio is comprised of two approved products and multiple clinical and preclinical product candidates. Approved products include Naglazyme(R) (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin, and Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation. Investigational product candidates include Kuvan(TM) (sapropterin dihydrochloride), a Phase 3 product candidate for the treatment of phenylketonuria (PKU), and 6R-BH4 for cardiovascular indications, which is currently in Phase 2 clinical development for the treatment of peripheral arterial disease and sickle cell disease. Both products are being developed in partnership with Merck Serono, a division of Merck KgA of Darmstadt, Germany. For additional information, please visit http://www.bmrn.com/. Information on BioMarin's website is not incorporated by reference into this press release.
About AnGes MG, Inc.
AnGes MG, Inc., a biopharmaceutical company founded in 1999, was established on an innovative discovery by researchers of Osaka University. The company specializes in research, development and practical application of genetic medicine. Current programs include the Hepatocyte Growth Factor (HGF) genetic medicine which improves blood circulation by regenerating blood vessels, and an NFkB decoy that controls various types of inflammation.
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the potential approval of Naglazyme in Japan; the continued clinical development and commercialization of Naglazyme; actions by regulatory authorities. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: our success in the continued commercialization of Naglazyme; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning each of the described products and product candidates; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's 2006 Annual Report on Form 10-K, as amended, and the factors contained in BioMarin's reports on Form 10-Q and Form 8-K. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin(R) and Naglazyme(R) are registered trademarks of BioMarin Pharmaceutical Inc.
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