Naglazyme Approved by Japanese Ministry of Health
"We are proud to work with AnGes in bringing the first drug treatmentoption to MPS VI patients in Japan," said Stephen Aselage, Senior VicePresident of Global Commercial Development at BioMarin. "We are dedicated toproviding life-altering therapies to patients around the world and continue toexpand our geographic footprint through our patient identification andcommercialization efforts."
BioMarin established a marketing and distribution agreement with AnGes inDecember 2006, through which AnGes obtained exclusive rights to marketNaglazyme in the Japanese market. AnGes submitted a marketing application tothe MHLW in August 2007. Naglazyme was approved by the U.S. Food and DrugAdministration (FDA) in May 2005 and by the European Commission (EC) inJanuary 2006. As the first drug approved for MPS VI, the FDA and EC have bothdesignated Naglazyme as an orphan drug, conferring seven years of marketexclusivity in the United States and 10 years of market exclusivity in theEuropean Union. In addition, Naglazyme obtained orphan designation in June2007 from the 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 theaccumulation of certain complex carbohydrates, glycosaminoglycans (GAGs), inthe lysosomes, giving rise to progressive cellular, tissue and organ systemdysfunction. The majority of individuals with MPS VI die from disease-relatedcomplications between childhood and early adulthood. Additional informationcan be found at http://www.mpsvi.com.
Naglazyme is the first and only enzyme replacement therapy indicated forthe treatment of MPS VI. 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 respiratoryinfections, abdominal pain, diarrhea, ear pain, cough, and otitis media.Severe reactions included angioneurotic edema, hypotension, dyspnea,bronchospasm, respiratory distress, apnea, and urticaria. The most commonsymptoms 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 for adverse events and all patients who completed thedouble-blind portion of the trial continued to receive weekly infusions ofNaglazyme. Nearly all patients developed antibodies as a result of treatment,but the level of the immune response did not correlate with the severity ofadverse events. Because antihistamine use may increase the risk of apneicepisodes, evaluation of airway patency should be considered prior to theinitiation of treatment. Consideration to delay Naglazyme infusion should begiven when treating patients who present with an acute febrile or respiratoryillness. Additional information can be found at http://www.naglazyme.com.
BioMarin develops and commercializes innovative biopharmaceuticals forserious diseases and medical conditions. The company's product portfoliocomprises three approved products and multiple clinical and preclinicalproduct candidates. Approved products include Naglazyme(R) (galsulfase) formucopolysacchari
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