Nitisinone is now officially licensed to treat AKU in the European Union. In the UK, patients who attend the National AKU Centre are prescribed 2mg daily. Clinical research has shown that that nitisinone reduced HGA levels by up to 95%. From 2013, We ran a series of international clinical trials called DevelopAKUre, that measured the effectiveness of nitisinone in treating AKU. In 2020 we found out that this trial was successful and showed that the drug markedly reduced HGA levels in the body.
AKU is caused by a missing enzyme, which means the body cannot break down a substance called homogentisic acid (HGA). Nitisinone acts by blocking the enzyme which makes HGA. This then stops HGA from building up in the body and causing harm.
When a patient attends the National AKU Centre, they will receive a patient information booklet about nitisinone, containing all they need to know about the drug. Prof. L Ranganath & Dr M Khedr, leading consultants on AKU at the NAC, will speak to patients on a one-on-one basis about the drug and possible side effects that may occur.