Triheptanoin - Ultragenyx

Drug Profile

Triheptanoin - Ultragenyx

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

Latest Information Update: 14 Nov 2017

Price : *
* Final gross price and currency may vary according to local VAT and billing address.

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

  • 02 Nov 2017 Ultragenyx Pharmaceutical intends to conduct discussions with regulators regarding planned phase III trial in Lipid metabolism disorders by end of 2017
  • 09 Oct 2017 University of Texas Southwestern Medical Center plans a phase I trial for De-Vivo disease (NCT03301532)
  • 20 Sep 2017 Ultragenyx Pharmaceutical completes a phase II trial in De Vivo disease (In adolescents, In children, In adults, In infants) in USA, Australia, France, Israel, Italy, Spain, Hungary, and the United Kingdom (PO) (NCT01993186)
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at so we can help.

Back to top