Triheptanoin - Ultragenyx

Drug Profile

Triheptanoin - Ultragenyx

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

Latest Information Update: 10 Sep 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

  • 13 Jun 2017 University of Texas Southwestern Medical Center and National Institute of Neurological Disorders and Stroke plan a phase II trial for De Vivo disease (In children, In adolescents) in USA (NCT03181399)
  • 08 Mar 2017 Ultragenyx Pharmaceutical initiates enrolment in a phase I trial for De Vivo disease (In children) in USA (NCT03041363)
  • 01 Mar 2017 Sheba Medical Center in collaboration with Ultragenyx Pharmaceutical plans a phase II trial for Rett syndrome (NCT03059160)
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