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news.The Australian Therapeutic Goods Administration (TGA) has granted approval of the registration for BioMarin Pharmaceutical's Vimizim (elosulfase alfa) to treat patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome.
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The company said that Vimizim was reviewed under the Orphan Drug program and it is the first treatment approved in Australia for Morquio A syndrome, an ultra-rare, severely debilitating disease affecting about 3,000 patients in the developed world.
Australia MPS & Related Diseases Society National manager Nicole Millis said: "Treatment for Morquio A syndrome has been lagging behind for a number of years, leaving patients with few options that could really make a difference in their daily lives.
"We are grateful for BioMarin’s commitment to the MPS community and for providing a therapy to these patients."
In February, the US Food and Drug Administration (FDA) approved the Vimizim license application to treat patients with Morquio A syndrome.
Vimizim is also approved in Canada and the European Union, while marketing applications have been submitted in several other countries.
BioMarin chief medical officer Hank Fuchs said: "The approval of Vimizim in Australia underscores our relentless commitment to providing this much needed therapy to patients with Morquio A syndrome across the globe.
"We will continue to leverage our expertise and dedicate our resources to advancing therapies within the MPS community, as well as other communities with ultra-rare conditions, to ensure patients get the treatments they deserve."