Long-term efficacy and safety of once-monthly pasireotide in Cushing's disease: A Phase III extension study

Abstract

Objectives Many patients with Cushing's disease (CD) require chronic pharmacotherapy to control their hypercortisolism. We evaluated the efficacy and safety of long-acting pasireotide during a long-term extension study in patients with CD. Design Open-label extension to a 12-month Phase III study of long-acting pasireotide in CD (N = 150; NCT01374906). Patients Patients with mean urinary free cortisol (mUFC) = 12 months' treatment during the extension and could transit to a separate pasireotide safety study). mUFC was = 6.5%, FPG >= 7.0 mmol/L, antidiabetic medication use, or history of diabetes) at extension baseline and last assessment. Conclusions Long-acting pasireotide provided sustained biochemical and clinical improvements, with no new safety signals emerging, supporting its use as an effective long-term therapy for CD.