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