BioCentury
ARTICLE | Product Development

Making of Mepsevii

Why Ultragenyx developed Mepsevii for ultra-rare indication MPS VII

December 8, 2017 6:13 PM UTC

It may take a few years for Ultragenyx Pharmaceutical Inc. to recoup its investment in Mepsevii vestronidase alfa-vjbk, but the company expects to continue to reliably churn out treatments for ultra-rare diseases alongside a portfolio of products for larger Orphan indications.

FDA approved Mepsevii on Nov. 15 to treat mucopolysaccharidosis VII (MPS VII; Sly syndrome), an indication with only 200 patients worldwide...

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Ultragenyx Pharmaceutical Inc.