EMA Recommends New Treatment for Cushing's Disease

Lara C. Pullen, PhD

January 23, 2012

January 23, 2012 — The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use has recommended approval of pasireotide (Signifor, Novartis) as a treatment for patients with Cushing's disease who cannot have surgery, or for those for whom surgery has been unsuccessful.

"The recommendation to approve Signifor (pasireotide) for the treatment of Cushing’s disease is the first step in making a medicine available to European patients that has been studied in the indication and for which doctors and patients are provided with specific information about its use," according to an EMA news release.

Pasireotide is a somatostatin analogue that attaches to somatostatin receptors in the pituitary and block the release of adrenocorticotrophin. In clinical trials, pasireotide was shown to reduce the levels of cortisol in the urine by at least 50% in 41% of patients treated with a 900-μg dose, and in 34% of patients treated with a 600-μg dose.

Pasireotide is marketed as a solution for injection at 0.3, 0.6, and 0.9 mg. Its benefits are a result of its different binding pattern to the somatostatin receptor compared with that of the currently available somatostatin analogues. The most common adverse effects of pasireotide are hyperglycemia (including the development of diabetes mellitus) and diarrhea.

Pasireotide was designated as an orphan medicine by the EMA on October 8, 2009. Designation of orphan medicine status is based upon the seriousness of the condition; the existence of alternative methods of diagnosis, prevention, or treatment; and either the rarity of the condition or insufficient returns on investment. Cushing's disease affects approximately 0.4 in 10,000 people in the European Union.

Cushing's disease is a debilitating and life-threatening disease that is characterized by an excess of cortisol in the blood. It is caused by the presence of a small tumor of the pituitary gland, which makes too much adrenocorticotrophin. The first-line therapy for patients with Cushing's disease is surgical removal of the tumor, and surgery is sometimes followed by radiotherapy.