BioMarin submits Vimizim BLA

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) submitted a BLA to FDA for Vimizim elosulfase alfa (formerly GALNS) to treat mucopolysaccharidosis IVA (MPS-IVA, Morquio's syndrome). The company plans to submit a regulatory application in Europe by the end of the month for the recombinant human N-acetylgalactosamine-6-sulfatase. At a December R&D day, BioMarin said it plans to price Vimizim between $250,000-$400,000 per year, and that the molecule could potentially double the company's revenues, which were $500.7 million in 2012 (see BioCentury Extra, Dec. 6, 2012). ...