FDA approves BioMarin's Vimizim

FDA approved a BLA from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) for Vimizim elosulfase alfa to treat mucopolysaccharidosis IVA (MPS-IVA, Morquio's syndrome). The company said it immediately launched the recombinant human N-acetylgalactosamine-6-sulfatase, but said pricing information is not yet available. According to FDA, Vimizim is the first approved treatment for Morquio's syndrome, a rare inherited lysosomal storage disease that the agency said affects about 800 people in the U.S.

With Vimizim's approval, BioMarin also became the first company to receive a voucher under FDA's Rare Pediatric Disease Priority Review voucher program, which was created by PDUFA V to encourage the development of new drugs and biologics to prevent and treat rare pediatric diseases. The transferrable voucher entitles the holder to obtain Priority Review of any subsequent NDA or BLA that otherwise would receive a standard review (see BioCentury, Oct. 1, 2012). ...