Triheptanoin - Ultragenyx

Drug Profile

Triheptanoin - Ultragenyx

Alternative Names: C7 fatty acid; C7 oil; Glycerol triheptanoate; Glyceryl triheptanoate; UX-007

Latest Information Update: 07 Dec 2016

Price : $50

At a glance

  • Originator Baylor Research Institute; University of Queensland
  • Developer Emory University; INSERM; Oregon Health & Science University; Ultragenyx Pharmaceutical; University of British Columbia; University of Pittsburgh
  • Class Triglycerides
  • Mechanism of Action Fatty acid replacements; Triglyceride replacements
  • Orphan Drug Status

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

    Yes - De Vivo disease; Lipid metabolism disorders
  • 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

  • Phase II De Vivo disease; Huntington's disease; Lipid metabolism disorders; Rett syndrome
  • Preclinical Glycogen storage disease type V

Most Recent Events

  • 01 Nov 2016 Ultragenyx Pharmaceutical and University of Pittsburgh initiates a clinical trial for De Vivo disease (In adolescents, In adults, In children) in USA (PO) (NCT02968953)
  • 28 Sep 2016 Preclinical trials in Glycogen storage disease type V in USA (PO) before September 2016
  • 28 Sep 2016 The Institut National de la Santé Et de la Recherche Médicale, France and Rigshospitalet plan a phase II trial for Glycogen storage disease type V in France and Denmark (PO) (NCT02919631)
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