Vestronidase alfa - Ultragenyx Pharmaceutical

Drug Profile

Vestronidase alfa - Ultragenyx Pharmaceutical

Alternative Names: Mepsevii; Recombinant beta-glucuronidase - Ultragenyx; Recombinant human beta glucuronidase; rhGUS; UX-003; Vestronidase alfa - vjbk

Latest Information Update: 17 Nov 2017

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At a glance

  • Originator Saint Louis University
  • Developer Ultragenyx Pharmaceutical
  • Class Glucuronidases
  • Mechanism of Action Glucuronidase replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Mucopolysaccharidosis VII
  • New Molecular Entity Yes

Highest Development Phases

  • Registered Mucopolysaccharidosis VII

Most Recent Events

  • 15 Nov 2017 Registered for Mucopolysaccharidosis VII (In adolescents, In children, In adults) in USA (IV) - First global approval
  • 15 Nov 2017 Ultragenyx Pharmaceutical receives Rare Pediatric Disease Priority Review Voucher for treatment of Mucopolysaccharidosis VII
  • 23 May 2017 FDA assigns PDUFA action date of 16/11/2017 for vestronidase alfa for Mucopolysaccharidosis VII
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