BioMarin Pharmaceutical Inc. today announced the Australian Therapeutic Goods Administration granted approval of the registration of Vimizim (elosulfase alfa) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first treatment in Australia approved for this condition. Vimizim was reviewed under the Orphan Drug program.

Dr. Kaustuv Bhattacharya from The Children's Hospital at Westmead, who is currently using Vimizim on patients in a research trial said, "Morquio syndrome is a rare and difficult condition to have. VIMIZIM is the only definitive treatment available and offers the hope of better endurance to these patients in the future."

Morquio A syndrome is an ultra-rare, severely debilitating disease affecting an estimated 3,000 patients in the developed world. The disease occurs as a result of a deficiency of activity in an enzyme involved in glycosaminoglycan (GAG) metabolism. The pervasive and progressive accumulation of GAGs leads to significant morbidities and multisystemic clinical impairments resulting in diminished functional capacity, impaired quality-of-life and early mortality. The most common features of the disease are progressive skeletal dysplasia, the need for frequent surgical procedures related primarily to musculoskeletal or respiratory dysfunction, and significant limitations in mobility, endurance and breathing.

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