The European Commission has issued on 22 October 2020 the approval of the extended indication for Orfadin (nitisinone) to include treatment of adult patients with rare disease alkaptonuria (AKU).
This was possible through the positive results of a four year clinical trial of nitisinone in AKU that was funded by the European Commission. The trial has shown excellent decrease in homogentisic acid (the cause of AKU), a significant reversal of the disease process (called ochronosis) and further benefits.
Story: Efficacy and safety of once-daily nitisinone for patients with alkaptonuria (SONIA 2): an international, multicentre, open-label, randomised controlled trial
- Publication date
- 26 October 2020
- Directorate-General for Research and Innovation