Triheptanoin - Ultragenyx

Drug Profile

Triheptanoin - Ultragenyx

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

Latest Information Update: 24 May 2018

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At a glance

  • Originator Baylor Research Institute; University of Queensland
  • Developer Emory University; INSERM; Oregon Health & Science University; Rigshospitalet; 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 Glycogen storage disease type V; Huntington's disease; Lipid metabolism disorders; Rett syndrome

Most Recent Events

  • 08 May 2018 Copenhagen Neuromuscular Center plans a phase II trial for Glycogen-storage-disease in Denmark , (EudraCT2017-004153-17)
  • 07 May 2018 Ultragenyx completes enrolment in its phase III trial for De Vivo disease (In childeren, In adolescents, In adults) in USA, France, United Kingdom, Spain, Italy, Germany (NCT02960217)
  • 07 May 2018 Ultragenyx plans a phase III trial for Lipid metabolism disorders
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