Biotech SF: BioMarin shares rise on data from late-stage rare disease trial

Monday, November 5, 2012

BioMarin shares rise on data from late-stage rare disease trial

BioMarin CEO Jean-Jacques Bienaime.

BioMarin Pharmaceutical Inc. will seek regulatory approval early next year of a drug to treat a crippling, inherited disease, after a late-stage clinical trial showed that its hit its goal of helping patients walk farther, the company said Monday.

On the news, shares of the Novato-based drug developer (NASDAQ: BMRN) jumped 29 percent, or $10.84 per share, through mid-afternoon trading to $48.25.
Although the disease -- called Mucopolysaccharidosis Type IVA, or MPS IVA -- affects just 3,000 patients, so-called orphan disease drug developers can charge six figures for the treatments. What's more, insurers generally will cover the cost of drugs that significantly improve or save patients' lives.

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