Image of the Orfadin Nitisinone label


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.¹ Clinical research has shown that that nitisinone reduced HGA levels by up to 95%.² 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.³ 

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.



Nitisinone access for International Patients

A number of international AKU sister societies have been established who can provide support for accessing nitisinone around the world. Contact information for these groups can be found here.

Cycle Pharmaceuticals and Inceptua Group have partnered to expand their Free Goods Programme to allow eligible AKU patients to access nitisinone. This programme makes nitisinone available to patients who either have no alternative treatment option or have exhausted all other treatment options in their country of residence.

More information on this programme and eligible countries can be found here.

If you require any assistance in accessing nitisinone treatment, please contact