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Vimizim Marketing Application Submitted To ANVISA In Brazil

SAN RAFAEL, Calif., July 1, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that it has submitted the Vimizim Marketing Application (MA) to the Agência Nacional de Vigilancia Sanitaria (ANVISA), or the National Health Surveillance Agency Brazil. Assuming priority review status, based on orphan drug status in the U.S. and EU, an approval decision from ANVISA is anticipated in mid 2014.

"Considering that Brazil, along with the U.S., are expected to be the largest market opportunities for Vimizim, the timely submission in this market is an important milestone in our efforts to bring the first therapeutic option to patients with Morquio A Syndrome," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "Along with orphan drug designation in the U.S. and EU, priority review status in the U.S. and accelerated assessment status in the EU, we are making good progress on a global basis. In the coming months, we will continue to work closely with regulatory authorities and prepare for the launch of Vimizim worldwide."


Mucopolysaccharidosis IVA (MPS IVA, also known as Morquio A Syndrome) is a disease characterized by deficient activity of N-acetylgalactosamine-6-sulfatase (GALNS) causing excessive lysosomal storage of glycosaminoglycans such as keratan sulfate and chondroitin sulfate. This excessive storage causes a systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance. Malformation of the chest impairs respiratory function, and looseness of joints in the neck cause spinal instability and potentially spinal cord compression. Other symptoms may include hearing loss, corneal clouding, and heart disease. Initial symptoms often become evident in the first five years of life. The disease substantially limits both the quality and length of life of those affected.

The rate of incidence of MPS IVA is as yet unconfirmed and varies among different populations but estimates vary between 1 in 200,000 live births and 1 in 250,000 live births. The estimated prevalence is approximately 3,000 patients in the developed world. Based on knowledge of the worldwide distribution of the MPS VI market and the more than 1,300 identified MPS IVA patients worldwide, the company estimates that approximately 20 percent of patients are in North America (15 percent in the U.S.) and approximately 50 percent of patients are in EUMEA. 

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