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Triheptanoin - Ultragenyx Pharmaceutical

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Drug Profile

Triheptanoin - Ultragenyx Pharmaceutical

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

Latest Information Update: 12 Aug 2026

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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 - Glycogen storage disease type II; Lipid metabolism disorders; 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

  • 21 May 2026 Launched for Lipid metabolism disorders in Japan (PO)
  • 23 Mar 2026 Registered for Lipid metabolism disorders in Japan (PO)
  • 05 Nov 2024 Ultragenyx Pharmaceutical announces intention to submit NDA to PMDA for Lipid metabolism disorders in mid-2025

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