KUVAN (sapropterin) is a drug treatment for PKU which was licensed in 2008.  This drug is available across Europe but has never been widely used by the NHS.

Read a factsheet on Kuvan:

Kuvan Factsheet

Watch Amy and her son William talking about using KUVAN.

PALYNZIQ (pegvaliase-pqpz) Injection for PKU

Palynziq was licensed in 2019.

Palynziq is a medicine that is used to treat phenylketonuria (PKU) in adults and adolescents from 16 years of age.  Palynziq is used in patients whose blood levels of phenylalanine have not been adequately controlled with other treatments.

NSPKU have been informed that Palynziq will be appraised by NICE for use in the NHS following the collection of further data for the use in health technology assessments.

Future research

There are numerous ongoing clinical trials into potential new therapies for PKU.

What is NSPKU doing about accessing new treatments for PKU?

NSPKU wants people with PKU to be able to have the choice to access new treatments to manage their condition.

We have launched a campaign to put pressure on the Government, NHS and pharmaceutical companies to make these drugs available in the UK.  You can learn more about the campaign.  Get involved! Make your voice heard too.