Decision

Triheptanoin in the treatment of long-chain fatty acid oxidation disorders (LC-FAOD)

Triheptanoin used to treat long-chain fatty acid oxidation disorders (LC-FAOD), a group of rare inherited disorders, in adults and children of all ages.

• From: Medicines and Healthcare products Regulatory Agency
• Published: 8 April 2025
• Last updated: 13 April 2026 — See all updates

Documents

ANNEX to Public Assessment Report

PDF, 357 KB, 1 page

This file may not be suitable for users of assistive technology.

Request an accessible format.

If you use assistive technology (such as a screen reader) and need a version of this document in a more accessible format, please email webupdates@mhra.gov.uk. Please tell us what format you need. It will help us if you say what assistive technology you use.

Triheptanoin: Public Assessment Report (PAR)

PDF, 374 KB, 4 pages

This file may not be suitable for users of assistive technology.

Request an accessible format.

If you use assistive technology (such as a screen reader) and need a version of this document in a more accessible format, please email webupdates@mhra.gov.uk. Please tell us what format you need. It will help us if you say what assistive technology you use.

Triheptanoin: Treatment Protocol for Healthcare Professionals

PDF, 623 KB, 16 pages

This file may not be suitable for users of assistive technology.

Request an accessible format.

If you use assistive technology (such as a screen reader) and need a version of this document in a more accessible format, please email webupdates@mhra.gov.uk. Please tell us what format you need. It will help us if you say what assistive technology you use.

Triheptanoin: Treatment Protocol for Patients

PDF, 491 KB, 8 pages

This file may not be suitable for users of assistive technology.

Request an accessible format.

If you use assistive technology (such as a screen reader) and need a version of this document in a more accessible format, please email webupdates@mhra.gov.uk. Please tell us what format you need. It will help us if you say what assistive technology you use.

Triheptanoin: Treatment Protocol on the Pharmacovigilance System

PDF, 145 KB, 5 pages

This file may not be suitable for users of assistive technology.

Request an accessible format.

If you use assistive technology (such as a screen reader) and need a version of this document in a more accessible format, please email webupdates@mhra.gov.uk. Please tell us what format you need. It will help us if you say what assistive technology you use.

Triheptanoin: Information for NHS Medical Directors

PDF, 472 KB, 4 pages

This file may not be suitable for users of assistive technology.

Request an accessible format.

If you use assistive technology (such as a screen reader) and need a version of this document in a more accessible format, please email webupdates@mhra.gov.uk. Please tell us what format you need. It will help us if you say what assistive technology you use.

Details

The renewed scientific opinion was issued on the 07 April 2026 to Ultragenyx Netherlands B.V for Triheptanoin in the treatment of paediatric and adult patients with long-chain fatty acid oxidation disorders (LC-FAOD).

The renewed scientific opinion includes

• a public assessment report including annex (updated 07 April 2026)
• a treatment protocol:
  • for healthcare professionals
  • for patients
  • on the pharmacovigilance system
• Information for NHS Medical Directors

Published 8 April 2025

Last updated 13 April 2026 + show all updates

1. 13 April 2026

   Renewal of EAMS scientific opinion issued to Ultragenyx Netherlands B.V for Triheptanoin in the treatment of paediatric and adult patients with long-chain fatty acid oxidation disorders (LC-FAOD), including the annex to the public assessment report.

2. 8 April 2025

   First published.