BioMarin’s Vimizim OK’d in Japan for Morquio A Syndrome

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BioMarin Pharmaceutical Inc. (BMRN) announced that the Japanese Ministry of Health, Labour and Welfare has approved the company’s enzyme replacement therapy, Vimizim, for the treatment of patients suffering from mucopolysaccharidosis type IVA or Morquio A syndrome.

Vimizim was reviewed by the Japanese regulatory authority under the orphan drug program. Following the approval, Vimizim becomes the first therapy to be cleared in the country for the treatment of this rare genetic disorder that is estimated to affect 3,000 people in the developed world, as per BioMarin.

We note that Vimizim is already approved and marketed in the U.S. and EU for the same indication. Vimizim generated sales of $40.4 million in the first nine months of 2014. The company expects Vimizim sales in the range of $65 million to $70 million for 2014. Apart from the U.S. and EU, Vimizim is also approved in Australia, Canada and Brazil. Approval in additional markets will boost the drug’s sales potential further.

Meanwhile, Biomarin is set to acquire the Dutch biotechnology company, Prosensa (RNA), in a cash transaction valued at about $680 million or $17.75 per share. The acquisition will add drisapersen, a late-stage Duchenne muscular dystrophy candidate, to BioMarin’s rare-disease portfolio. The deal is slated to go through in the first quarter of 2015 (read more: Prosensa Soars on $680M Buyout Offer from BioMarin).

BioMarin currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Acorda Therapeutics, Inc. (ACOR) and ANI Pharmaceuticals, Inc. (ANIP). Both stocks carry a Zacks Rank #1 (Strong Buy).

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