In September 2020, Swedish Orphan Biovitrum AB announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion for Orfadin (nitisinone) for the treatment of adult patients with alkaptonuria (AKU), which was the first described human genetic disease. The opinion has been given based on the scientific results of the DevelopAKUre clinical development programme, a research consortium initiated by the AKU Society and clinical experts. The DevelopAKUre programme included a long-term, international phase 3 clinical efficacy study in 138 patients demonstrating that nitisinone 10 mg per day was well tolerated and shown to be effective in reducing urinary excretion of the causal agent of alkaptonuria, homogentisic acid (HGA), resulting in a reduction of the disease process (ochronosis) and clinical signs, indicating a slower disease progression in adult patients
