A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7

Trial Profile

A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7

Completed
Phase of Trial: Phase III

Latest Information Update: 23 May 2017

At a glance

  • Drugs Vestronidase alfa (Primary)
  • Indications Mucopolysaccharidosis VII
  • Focus Registrational; Therapeutic Use
  • Sponsors Ultragenyx Pharmaceutical
  • Most Recent Events

    • 23 May 2017 The Prescription Drug User Fee Act (PDUFA) goal date for a decision is 16 November 2017 and an opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected in the first half of 2018.
    • 23 May 2017 According to an Ultragenyx Pharmaceutical media release, the Biologics License Application (BLA) and the Marketing Authorization Application (MAA) of the recombinant human beta-glucuronidase (rhGUS, UX003), submitted to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) respectively, for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome) were accepted for review.
    • 06 Mar 2017 Status changed from active, no longer recruiting to completed.
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