BioMarin Prices New Drug For Ultra-Rare Disease at $380K Per Year

BioMarin Pharmaceuticals (BMRN) is pricing its latest orphan disease drug Vimizim at $380,000 per year, including discounts, the company announced Tuesday. 

The FDA approved Vimizim on Friday for the treatment of Morquio A Syndrome, a very rare genetic disorder which causes problems with bone development, growth and mobility. BioMarin has identified approximately 1,500 Morquio A patients worldwide, with about 15% of them in the U.S.

European drug regulators are expected to render an approval opinion on Vimizim Friday. 

BioMarin expects Vimizin sales in the range of $60-70 million in 2014 with more than 350 patients on therapy by the end of the year. 

The company's total revenue guidance for 2014 is $650-680 million, or 20% growth over 2013. Current consensus has BioMarin generating $670 million in total revenue. 

The biggest knock on BioMarin is that new drug approvals and sales growth have not flowed appreciably to the bottom line where the company continues to lose money. This year won't be different, with BioMarin guiding to higher R&D expenses and a non-GAAP net loss of $100-130 million range. 

BioMarin believes 2014 will be a peak year for R&D expenses as a percentage of sales with the metric falling in 2015 and beyond. The company reiterated guidance that profitability will be achieved once total revenue reaches $1 billion, which could happen based on Vimizin peak sales opportunity. 

Key phase III data on a longer-acting version of BioMarins drug for PKU are expected in the fourth quarter. 


Adam Feuerstein writes regularly for TheStreet. In keeping with company editorial policy, he doesn't own or short individual stocks, although he owns stock in TheStreet. He also doesn't invest in hedge funds or other private investment partnerships. Feuerstein appreciates your feedback; click here to send him an email.

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