Triheptanoin - Ultragenyx

Drug Profile

Triheptanoin - Ultragenyx

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

Latest Information Update: 14 Nov 2017

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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
  • New Molecular Entity Yes

Highest Development Phases

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

Most Recent Events

  • 02 Nov 2017 Ultragenyx Pharmaceutical intends to conduct discussions with regulators regarding planned phase III trial in Lipid metabolism disorders by end of 2017
  • 09 Oct 2017 University of Texas Southwestern Medical Center plans a phase I trial for De-Vivo disease (NCT03301532)
  • 20 Sep 2017 Ultragenyx Pharmaceutical completes a phase II trial in De Vivo disease (In adolescents, In children, In adults, In infants) in USA, Australia, France, Israel, Italy, Spain, Hungary, and the United Kingdom (PO) (NCT01993186)
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