AKU can be treated using the drug nitisinone. Our seven-year DevelopAKUre study, which concluded in 2020, illustrated that nitisinone effectively halts the build-up of homogentisic acid (HGA) by 99%.
Nitisinone received a marketing authorisation from the European Medicines Agency (EMA) and is now available to patients across Europe. Patients in the UK can access nitisinone treatment by attending the National AKU Centre (NAC).
In June 2025, the U.S. Food and Drug Administration (FDA) approved HARLIKU™ (nitisinone) tablets for adults with AKU – the first and only FDA-approved treatment for AKU in the United States.
AKU can also be treated through a number of self-management techniques including exercise and pain management.
Nitisinone
Nitisinone is a life-changing treatment which reduces the acid which causes AKU by 99%...
Diet
Patients who are not taking nitisinone should not change their diet without consulting their doctor...
