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Vestronidase alfa - Ultragenyx Pharmaceutical

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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: 05 Nov 2023

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At a glance

  • Originator Saint Louis University
  • Developer Amicus Therapeutics; 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

  • 31 Dec 2021 Preregistration for Mucopolysaccharidosis VII (In adolescents, In children, In adults) in Mexico (IV) prior to December 2021
  • 31 Dec 2021 Registered for Mucopolysaccharidosis VII (In adolescents, In children, In adults) in Mexico (IV) prior to December 2021
  • 09 Sep 2021 Launched for Mucopolysaccharidosis VII in Italy (IV) before September 2021

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