SAN RAFAEL, Calif., May 30, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for Vimizim (BMN-110, elosulfase alfa), an enzyme replacement therapy under evaluation for the treatment of patients with the rare lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome. The FDA has granted priority review designation to Vimizim, which is granted to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists. During the initial review of the application, the FDA requested additional Chemistry, Manufacturing and Controls (CMC) information. The company provided the information as requested, and the FDA designated it as a major amendment to the application thus extending the PDUFA action date by three months. The extended PDUFA action date is February 28, 2014. In the FDA's filing communication, the Agency informed the company that it is currently planning to hold an advisory committee meeting to discuss the application. No date has been set for this meeting. "We are pleased that the FDA has granted priority review to the Vimizim BLA, and we look forward to a productive dialog with the FDA as they review the application," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "With our expertise in developing enzyme replacement therapies to treat serious unmet medical needs, we plan to work closely with the FDA and other regulatory authorities to bring this much needed therapy to MPS IVA 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.