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Vimizim MAA Validated By The EMA

SAN RAFAEL, Calif., May 30, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for Vimizim. Validation of the MAA confirms that the submission is complete and starts the EMA's formal review process. Earlier this year, the EMA accepted BioMarin's request for accelerated assessment for this MAA on the grounds that Vimizim could satisfy an unmet medical need and is of major interest from the point of view of therapeutic innovation and public health. Accelerated assessment has the potential to shorten EMA's review procedure. However, at any time during the MAA assessment, the EMA may decide to continue the assessment under standard assessment timelines. Assuming the Vimizim application remains on the accelerated assessment timeline, a CHMP opinion is anticipated in December 2013, and, if positive, a decision from the European Commission could be received in the first quarter of 2014.

"With approximately half of all MPS IVA patients living in Europe, the Middle East and Africa, this 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 the acceptance of the BLA by the FDA, we are pleased to enter the review phase on a global basis. MPS IVA represents a significant unmet medical need for those affected, and our hope is to offer a life-altering treatment option to patients worldwide."

About MPS IVA

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.

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