FDA approves Ultragenyx's Mepsevii for MPS VII

FDA approved Mepsevii vestronidase alfa-vjbk (rhGUS, UX003) from Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) to treat mucopolysaccharidosis VII (MPS VII; Sly syndrome). Mepsevii is the first therapy approved for the indication.

Ultragenyx said Mepsevii will be available to patients in the U.S. this month. The company did not disclose a price for the therapy, but said it will be priced in line with enzyme replacement therapies (ERTs) for other MPS disorders...