Nitisinone




Patients who attend the National AKU Centre are given off-license nitisinone.

Nitisinone is currently licensed for use in treating another similar rare disease called hereditary tyrosinemia type 1 (HT-1). Patients who attend the National AKU Centre are prescribed 2mg daily, a significantly lower dose than prescribed in treating HT-1. Although it is not currently licensed for use in AKU, previous animal experiments and research studies have shown that nitisinone could be effective in treating it. Clinical research in the US showed that nitisinone reduced HGA levels by up to 95%. We are currently running an international clinical trial called DevelopAKUre, measuring the effectiveness of nitisinone in treating AKU.

AKU is caused by a missing enzyme, which means the body cannot break down a substance called homogentisic acid (HGA). Nitisinone acts by stopping the enzyme which makes HGA from working. 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, the leading doctor and consultant at the centre, will speak to patients on a one on one basis about the drug and any possible side effects that may occur. 

 

Published: 24.07.16 Next Review Date: 24.07.19





AKU Twitter

AKU Blog

Meet the patient - Simon Laxon
18-Apr-2019

Today’s blog is written by AKU patient Simon Laxon. In it, he writes about .. ...more

Stand Up To AKU 2019!
12-Apr-2019

We are excited to announce that our Stand up to AKU Comedy Night is back! This year we are h.. ...more

Luton Health Awareness Event
05-Apr-2019

On Tuesday the 26th of March, our Patient Support Manager, Lesley, and Fundraising Officer, .. ...more

EURORDIS Winter School 2019
29-Mar-2019

In today's blog, CEO and Chair Nick Sireau tells us about his recent trip to Paris for t.. ...more

Rare Disease Day 2019
14-Mar-2019

Rare Disease Day is an international day designed to raise awareness to the public and those.. ...more