The Medicines & Healthcare products Regulatory Agency (MHRA), an executive agency of the Department of Health and Social Care in the UK, which is responsible for ensuring that medicines work and are acceptably safe, has published a positive opinion to make triheptanoin available to the LC-FAOD community in the UK. 

The MHRA can provide early availability to promising new unlicensed medicines to UK patients that have a high unmet need. Triheptanoin has fulfilled MHRA’s criteria and will therefore be made available. 

Please note that Ultragenyx has to activate the scheme, after which your clinician can apply for access. It is expected that this will take a few months. We will share an update once we have a more concrete timeline. 

In the meantime, please read our full update via the button below, including information about the formal assessment of triheptanoin for inclusion by the NHS in the UK: