Pasireotide

Patients with Cushing's disease in whom surgical removal of the pituitary adenoma is not an option have few medical alternatives.The few available drugs are poorly evaluated and are used off-licence. Pasireotide, an injectable somatostatin analogue, is the first drug to be authorised in the EU for these patients. Clinical evaluation is based on one trial evaluating two doses of pasireotide and two uncontrolled trials. Average urinary free cortisol levels normalised in about 25% of cases. In one trial, almost all of the patients had adverse effects (severe in about 25% of cases) attributed to pasireotide: hyperglycaemia, diarrhoea, nausea, gallstones, QT prolongation and bradycardia. In practice, pasireotide has a marginally favourable harm-benefit balance and has not been compared with the principal drugs used (off-licence) in Cushing's disease. However, when surgery fails or is not possible, the use of pasireotide may sometimes be justified, provided that patients are closely monitored.