BioMarin prices Vimizim, reports preliminary results

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) reported preliminary 4Q13 and 2013 financial results on Tuesday and disclosed the price of newly launched Vimizim elosulfase alfa, which FDA approved on Friday for mucopolysaccharidosis IVA (MPS-IVA, Morquio's syndrome). BioMarin said a 5 mg vial of the recombinant human N-acetylgalactosamine-6-sulfatase is available with a wholesale acquisition cost (WAC) of $1,068. The recommended dose is 2 mg/kg once weekly. Including "discounts and expected compliance," the company said this translates to an average annual cost of about $380,000 for a typical 22.5 kg patient. An MAA for the enzyme replacement therapy is under review in the EU; the company said it expects an opinion from EMA's CHMP on Friday. BioMarin reiterated that it expects 2014 Vimizim sales of $60-$70 million (see BioCentury Extra, Feb. 14).

BioMarin said it expects to report 4Q13 revenues of $146.9 million and 2013 revenues of $548.5 million. The Street was expecting revenues for the quarter of $143.3 million and $545.9 million for the year. The company reaffirmed that it expects 2013 non-GAAP net loss of $40-$60 million. BioMarin also reiterated its 2014 revenue guidance of $650-$680 million. The company plans to release full financial results next week. ...