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An Open-label Phase 2 Study to Assess Safety and Clinical Effects of UX007 in Subjects With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Trial Profile

An Open-label Phase 2 Study to Assess Safety and Clinical Effects of UX007 in Subjects With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 30 Oct 2019

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At a glance

  • Drugs Triheptanoin (Primary)
  • Indications Lipid metabolism disorders
  • Focus Therapeutic Use
  • Sponsors Ultragenyx Pharmaceutical
  • Most Recent Events

    • 14 Oct 2019 According to an Ultragenyx Pharmaceutical media release, the U.S. Food and Drug Administration (FDA) has accepted for review the companys New Drug Application (NDA) for UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD).The FDA has assigned a standard review designation with a Prescription Drug User Fee Act (PDUFA) target date of 31st Jul 2020.Data from this trial along with other studies supported the NDA filing.
    • 01 Aug 2019 According to an Ultragenyx Pharmaceutical media release, the company has submitted the New Drug Application (NDA) for UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD) to the U.S. FDA. Application is supported by data from this trial.
    • 19 Feb 2019 According to an Ultragenyx Pharmaceutical media release, the company is on track to submit a New Drug application (NDA) to the U.S. Food and Drug Administration (FDA) in mid-2019. Data from this trial along with other studies, will support the application.
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