SAN RAFAEL, Calif., April 1, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) 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 company intends to submit an application for registration in the European Union (EU) by the end of April 2013.
"Based on the positive results from our Phase 3 pivotal study, we believe that Vimizim offers a substantial benefit to patients with MPS IVA, a severely debilitating and progressive disease for which there is no current treatment," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin. "The submission of the BLA represents a significant milestone for BioMarin and is the result of the strong, collaborative effort of many hard working employees, investigators, patients, and their families. With this application, BioMarin continues in its long-standing tradition of developing important therapies for those who are most in need. We look forward to working with the U.S. regulatory authorities to bring this treatment to patients."
About MPS IVAMucopolysaccharidosis 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.