In November 2014 European Commission granted European marketing authorisation for Ketoconazole HRA® as an orphan drug for the treatment of Endogenous Cushing’s Syndrome. Ketoconazole-containing medicines has been used off-label to treat the syndrome for more than 30 years. Now Ketoconazole HRA® is a fully-authorised medicine which provides patients and their healthcare providers with evidence-based information for optimal patients’ management. Orphan designation carries a protection of 10 years of market exclusivity. The first launches of Ketoconazole HRA® in Europe are scheduled in the first quarter of 2015.
Endogenous Cushing’s syndrome is a disorder caused by prolonged exposure to high levels of the hormone cortisol. It is associated with increased mortality and multisystem morbidity. The syndrome is caused by tumors secreting adrenocorticotropin hormone (ACTH) whether from pituitary or extra-pituitary origin, cortisol, or very rarely corticotropin-releasing hormone. Definitive therapy of endogenous Cushing’s syndrome optimally involves tumor resection. Indications for medical therapy include acutely ill patients, preparation for surgery, those for whom surgery is not indicated, or patients who remain hypercortisolemic postoperatively or while awaiting the effectiveness of radiotherapy.