Success in DevelopAKUre: approval for Orfadin to treat patients with AKU

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.

More information

Commission decision

EU Community Register

Story: Efficacy and safety of once-daily nitisinone for patients with alkaptonuria (SONIA 2): an international, multicentre, open-label, randomised controlled trial