CSOM230B2410: Non-interventional study for the generation of long term safety and efficacy data of pasireotide s.c. in patients with Cushing's disease (Post-Authorization Safety Study)

Abstract

Introduction: With an annual incidence of 0.7 – 2.4 per million, Cushing's disease (CD) is a rare but devastating disease caused by an adrenocorticotropic hormone (ACTH) secreting pituitary adenoma. CD is associated with severe morbidity and mortality and most commonly affects adults aged 20 – 50, primarily females. Pituitary resection of the adenoma is the current first-line therapy for CD, but surgical failure rates are as high as 25 – 30%. Pasireotide (SOM230), a novel somatostatin analogue, exhibits a unique binding profile with high affinity to four of the five known human somatostatin receptor subtypes (sstr1, 2, 3 and 5) and is approved for pharmacological treatment of CD in the EU and U.S. for those patients for whom surgery has failed or is not an option. A phase III study [CSOM230B2305] showed efficacy of pasireotide s.c. in reducing and normalising hypercortisolism. However, since duration and patient numbers are limited, long-term effects could not be fully addressed.