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
Phase of Trial: Phase III
Latest Information Update: 15 Nov 2017
At a glance
- Drugs Vestronidase alfa (Primary)
- Indications Mucopolysaccharidosis VII
- Focus Registrational; Therapeutic Use
- Sponsors Ultragenyx Pharmaceutical
- 15 Nov 2017 According to an Ultragenyx Pharmaceuticals media release, the US FDA has approved MEPSEVII (vestronidase alfa) for the treatment of children and adults with Mucopolysaccharidosis VII (MPS VII, Sly syndrome).
- 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.
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Table of Contents
- At a glance
- Trial Overview
- Trial Details
- Trial Centres
- Trial History