Treatment & Clinical Trials
Explainer Video
View a short explainer video about alkaptonuria. In English. In French. In Spanish.
Nitisinone Treatment
Nitisinone is a potential treatment for AKU patients. Dr Duncan Batty, AKU patient, AKU volunteer, and former pharma industry researcher with a PhD in biochemistry, has put together a document designed to give AKU patients his viewpoint on what Nitisinone is, why it is a possible treatment for AKU and what is known about its long term effects.
Download it here: Taking-Nitisinone.pdf
HARLIKU™ (nitisinone) Tablets are the first and only FDA-approved treatment available in the U.S. for people living with alkaptonuria (AKU).
You can learn more about how HARLIKU works, the support available for patients and families, and how to enroll by visiting: HARLIKU™ (nitisinone) Tablets | Patients
Warnings & Precautions: Ocular symptoms including keratitis (inflammation of the cornea, eye irritation, eye pain, photophobia) and hyperkeratotic plaques (thick, rough skin) due to elevated plasma tyrosine levels, leukopenia (low number of white blood cells) and severe thrombocytopenia (low levels of platelets in the blood).
Most common side effects: Elevated tyrosine levels, keratitis and thrombocytopenia. If you have any new or worsening negative side effects while taking HARLIKU™ you should immediately call your healthcare provider.
Please refer to the full Prescribing Information at: harliku.com/pi/
Clinical Trials
SONIA 1 Published Results
The main scientific paper from the first study (SONIA 1) was published in the Annals of Rheumatic Diseases. This study was the first part of the ongoing clinical trial to find out what dose of nitisinone is best for patients. Read more
SONIA 2 Nitisinone Trial Results
We are delighted to announce that all the data from the SONIA 2 clinical trial has now been analyzed and very positive trends have been highlighted. Due to this, SOBI (Swedish Orphan Biovitrum) announced that they are going to apply to the European Medicines Agency (EMA) for market authorization for the drug to be used in treating AKU. If successful, this could lead to nitisinone being licensed and available to all AKU patients in Europe. Read more
Nitisinone Different Age Groups of Patients with Alkaptonuria
The study is designed to treat patients and find the optimal dosage of nitisinone to obtain maximal reduction in levels of homogentisic acid and maintain safe levels of tyrosine. The long term objective in the target population of pre-symptomatic patients is the prevention of the characteristic effects on joint cartilage and tendons. Read more