Research programme: enzyme replacements - Ultragenyx

Drug Profile

Research programme: enzyme replacements - Ultragenyx

Alternative Names: rhPPCA; UX 004

Latest Information Update: 25 Mar 2017

Price : $50

At a glance

  • Originator St. Jude Childrens Research Hospital
  • Developer Ultragenyx Pharmaceutical
  • Class Enzymes
  • Mechanism of Action Enzyme replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

  • 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.

  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Lysosomal storage diseases

Most Recent Events

  • 26 Feb 2016 Ultragenyx Pharmaceuticals announces intention to submit an IND for UX 004 in 2017 (Ultragenyx Pharmaceuticals, Form 10-K, February 2016)
  • 27 Mar 2015 Ultragenyx Pharmaceuticals in-licenses rhPPCA from St. Jude Children’s Research Hospital (Ultragenyx Pharmaceuticals form 10-K filed in March 2015)
  • 23 Sep 2013 Preclinical development of UX 004 is ongoing in USA
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