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news.BioMarin Pharmaceutical has announced that Health Canada has approved VIMIZIM(elosulfase alfa) for long-term enzyme replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis IVA (MPS IVA), also known as Morquio A syndrome.
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The approval makes VIMIZIM the first and only pharmaceutical treatment option available in Canada for children and adults living with this severely debilitating, progressive and life-limiting disorder.
"Morquio A is an ultra-rare disease for which there is no cure. In fact, until now, available treatments only addressed the symptoms of the disease," said Dr. John Mitchell , a leading pediatric endocrinologist and biochemical geneticist based in Montreal , and clinical investigator in the VIMIZIM Phase 3 trial.
"The approval of VIMIZIM fills a critical unmet need for Morquio A patients and their families as it moves treatment beyond supportive care to addressing the underlying cause of the disease."
The New Drug Submission for VIMIZIM was submitted to Health Canada in October 2013 under Priority Review Status, which allows for the "fast-tracking" of submissions intended for the treatment, prevention or diagnosis of serious, life-threatening or severely debilitating diseases or conditions.
"The Health Canada approval of VIMIZIM is a significant milestone for BioMarin, and for Canadians living with Morquio A and their families," said Hank Fuchs , M.D., Chief Medical Officer of BioMarin.
"To date, BioMarin has developed treatments for three different MPS diseases. The approval of VIMIZIM firmly establishes our leadership in advancing important therapies for the treatment of MPS diseases. We will continue to build on our extensive scientific and clinical knowledge of lysosomal storage disorders to develop therapies for other rare genetic diseases."