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Triheptanoin - Ultragenyx Pharmaceutical

Drug Profile

Triheptanoin - Ultragenyx Pharmaceutical

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

Latest Information Update: 04 Mar 2024

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At a glance

  • Originator Baylor Research Institute
  • Developer Ultragenyx Pharmaceutical
  • Class Esters; Small molecules; Triglycerides
  • Mechanism of Action Triglyceride replacements
  • Orphan Drug Status

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

    Yes - Lipid metabolism disorders; Glycogen storage disease type II; De Vivo disease
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Lipid metabolism disorders
  • Phase II Glycogen storage disease; Glycogen storage disease type V; Hemiplegia; Huntington's disease; Rett syndrome
  • Phase I/II Amyotrophic lateral sclerosis
  • No development reported Glycogen storage disease type I
  • Discontinued De Vivo disease

Most Recent Events

  • 31 Dec 2023 Ultragenyx Pharmaceutical has patent protection for triheptanoin in USA, Australia, Brazil, Canada, Israel, Korea, Malaysia, and Taiwan
  • 27 Dec 2022 Phase-III clinical trials in Lipid metabolism disorders (In adolescents, In children, In infants, In neonates) in Spain, Poland, Czech Republic (PO) (EudraCT2022-001539-10)
  • 31 Dec 2021 Launched for Lipid metabolism disorders (In adolescents, In children, In infants, In adults) in Canada (PO) prior to December 2021
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