Sonia 2 Nitisinone Trial Results

SONIA 2 and DevelopAKUre – Results

We are delighted to announce that all the data from the SONIA 2 clinical trial has now been analysed 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 authorisation for the drug to be used in treating AKU. If successful, this could lead to nitisinone being licenced and available to all AKU patients in Europe.

CEO and Chair of the AKU Society Nick Sireau said:

“I originally joined the AKU Society because my two sons were born with AKU. I refused to let my children grow up with the debilitating symptoms I read about on the internet. I dedicated my life to finding an effective treatment for this disease. I now see a future where this may be possible.

“I am immensely proud of the hard work and dedication of everyone involved in the DevelopAKUre clinical trials over the last seven years, and I would like to thank every one of them. I would especially like to thank the patients: without their selfless sacrifice and steadfastness over the last years; we would not be where we are today without them.

“Although this is the end of a long process, it is also the beginning of a new one. It may be years before we know if nitisinone will become available. But one thing is for certain; we are now one huge step closer to an effective disease-modifying treatment available to AKU patients everywhere.”

Chief Investigator of DevelopAKUre Prof Ranganath said:

“It is wonderful that all the hard work that has gone into the study and the sacrifices made by patients to take part has been worthwhile. A big thank you to everyone. ”

More information will follow shortly. If you would like more information please email ciaran@akusociety.org.