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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: 14 Aug 2019

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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

  • Marketed Mucopolysaccharidosis VII

Most Recent Events

  • 08 Aug 2019 Amicus Therapeutics announces intention to submit NDA to regulatory body in Japan for Mucopolysaccharidosis VII
  • 26 Mar 2019 Ultragenyx Pharmaceutical completes a phase II trial in Mucopolysaccharidosis VII (In children, In infants, In neonates) in USA, Spain and Portugal (NCT02418455)
  • 15 Jan 2019 Phase-II clinical trials in Mucopolysaccharidosis VII (In children, In adolescents, In adults) in Japan (IV) (JMA-IIA00416)
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