Vestronidase alfa

Drug Profile

Vestronidase alfa

Alternative Names: Recombinant beta-glucuronidase - Ultragenyx; Recombinant human beta glucuronidase; rhGUS; UX-003

Latest Information Update: 10 Sep 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

  • Preregistration Mucopolysaccharidosis VII

Most Recent Events

  • 23 May 2017 Preregistration for Mucopolysaccharidosis VII (In adolescents, In children, In adults) in European Union (IV)
  • 23 May 2017 Preregistration for Mucopolysaccharidosis VII (In children, In adults, In adolescents) in USA (IV)
  • 23 May 2017 An opinion from the Committee for Medicinal Products for Human Use for vestronidase alfa indicated for Mucopolysaccharidosis VII is expected in the first half of 2018
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