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news.Brazilian Health Surveillance Agency (Anvisa) has granted approval for US-based BioMarin Pharmaceutical's Vimizim (elosulfase alfa) to treat patients with mucopolysaccharidosis type IVA (MPS IVA) also known as morquio A syndrome, an ultra-rare genetic condition.
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Earlier this month, Vimizim was also approved in Australia for the treatment of this rare, severely debilitating and progressive disease that previously had no approved, standard-of-care treatment other than supportive care.
Vimizim is the first approved enzyme replacement therapy (ERT) designed to target the underlying cause of Morquio A Syndrome, a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS).
The drug is intended to provide the exogenous enzyme GALNS that will be taken up into the lysosomes and increase the catabolism of glycosaminoglycans (GAGs).
Brazil’s Federal University of Rio Grande do Sul professor of Genetics Roberto Giugliani said: "As a physician dedicated to the MPS community, I am delighted to have the opportunity to move my patients suffering from Morquio A syndrome beyond supportive care to a therapy that can address their condition at the cellular level and potentially change the course of the disease.
"The approval of Vimizim in Brazil is a significant milestone for the community of patients with Morquio A syndrome who can now receive for the first time a specific treatment for their rare condition."
Across the about 3,000 patients are affected by morquio A syndrome, which occurs as a result of a deficiency of activity in an enzyme involved in GAG metabolism.
BioMarin Latin America senior director of Medical Services Daniela Giovannetti said: "Approval in Brazil advances our vision of taking this much needed therapy worldwide a reality.
"We are grateful to the patients from all over Latin America for participating in the VIMIZIM clinical trials. Their participation is absolutely essential to the success of our development program around the world."
In February 2014, 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, Australia and the EU, while marketing applications have been submitted in several other countries.
Image: Apart from Brazil, Vimizim was also approved this December in Australia to treat morquio A syndrome. Photo: courtesy of Baitong333/ freedigitalphotos.net.