UX 003

Drug Profile

UX 003

Alternative Names: Recombinant beta-glucuronidase - Ultragenyx; Recombinant human beta glucuronidase; rhGUS; UX-003; UX003-rhGUS

Latest Information Update: 19 Nov 2016

Price : $50

At a glance

  • Originator Saint Louis University
  • Developer Saint Louis University; 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
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    Mucopolysaccharidosis VII
  • New Molecular Entity Yes

Highest Development Phases

  • Phase III Mucopolysaccharidosis VII

Most Recent Events

  • 07 Nov 2016 Ultragenyx was issued composition of matter and method of treatment patent for UX 003 in USA with validity upto 2035
  • 14 Jul 2016 Adverse events and efficacy data from a phase III trial in Mucopolysaccharidosis VII released by Ultragenyx
  • 05 Aug 2015 UX 003 receives Fast Track designation for Mucopolysaccharidosis VII [IV,Infusion] (In children, In infants, In adults) in USA
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