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New drug targets cortisol production in Cushing’s disease

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Pasireotide was effective at reducing the cortisol levels and improving associated signs and symptoms in patients with Cushing’s disease, according to data from a phase 3 trial that were presented at the 14^th Congress of the European Neuroendocrine Association.

Cushing’s disease is caused when the adrenal glands produce excess cortisol. Pasireotide (SOM230, Novartis) controls the secretion of excess cortisol, which is triggered by adrenocorticotropic hormones, which are secreted by a benign pituitary tumor. The drug currently has orphan drug status in the United States and Europe. There are no medical treatments approved for the disease, and other options, including surgery and radiotherapy, have a limited benefit.

The PASPORT-CUSHINGS trial included 162 patients who had moderate to severe persistent/recurrent or de novo Cushing’s disease and were ineligible for surgery. The patients were randomly assigned to pasireotide 600 mcg twice daily or 900 mcg twice daily. The primary endpoint was the normalization of urinary free cortisol levels, which are used to diagnose and monitor Cushing’s disease.

At 3 months, patients whose urinary free cortisol levels were higher than their baseline levels were unblinded, and they received an increased dose of pasireotide. The remaining patients continued their assigned double blind dose. After 6 months, the trial was open label.

At 6 months, the urinary free cortisol levels were normalized in 26.3% of the patients in the 900-mcg group, meeting the primary endpoint. The urinary free cortisol levels were normalized in 14.6% of the patients in the 600-mcg group, which did not meet the primary endpoint. Also at 6 months, the median urinary free cortisol in both groups declined by 47.9%. At 12 months, the median reduction in urinary free cortisol levels was 67.6% in the 600-mcg group and 62.4% in the 900-mcg group.

The reduced urinary free cortisol levels also led to an improvement in the clinical symptoms of Cushing’s disease and reduced blood pressure, cholesterol and BMI. The most common adverse effects were diarrhea, nausea and hyperglycemia. There currently are studies focusing on the optimal management of the hyperglycemia.

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