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Triheptanoin - Ultragenyx

Drug Profile

Triheptanoin - Ultragenyx

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

Latest Information Update: 20 Nov 2019

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At a glance

  • Originator Baylor Research Institute; University of Queensland
  • Developer Duke University; 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

  • Preregistration Lipid metabolism disorders
  • Phase II Glycogen storage disease type V; Huntington's disease; Rett syndrome
  • Phase I/II Amyotrophic lateral sclerosis
  • Phase I Glycogen storage disease type I
  • Discontinued De Vivo disease

Most Recent Events

  • 01 Nov 2019 Ultragenyx Pharmaceuticals terminates a extension phase II trial for De Vivo disease due to lack of efficacy (In infants, In children, In adolescents, In adults) in USA, Australia, Denmark, Spain and United Kingdom (PO) (NCT02599961)
  • 14 Oct 2019 FDA assigns PDUFA action date of 31/07/2020 for triheptanoin for Lipid metabolism disorders (In adolescents, In adults, In children, In infants) (PO)
  • 14 Oct 2019 US FDA accepts NDA for triheptanoin for Lipid metabolism disorders (In adolescents, In adults, In children, In infants) (PO) for review
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