Drug for Cushing's Disease Shows Efficacy in Phase 3 Trial

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An investigational drug called pasireotide is effective at reducing cortisol levels in patients with Cushing's disease, according to the results of a phase 3 trial.

Cushing's disease is caused by a benign tumor in the pituitary gland that secretes adrenocorticotropic hormone, which triggers the adrenal glands to produce excess cortisol. This can result in severe cardiovascular and metabolic-related illnesses or death. There are no approved medications to treat the disease. The drug has orphan drug designation for Cushing's disease in the United States and Europe.

In the study of 162 patients, 26% of subjects randomized to receive pasireotide at a dosage of 900 μg twice daily achieved normal levels of urinary free cortisol (UFC), according to the drug's developer, Novartis Pharmaceuticals of East Hanover, N.J. Reductions in UFC levels were accompanied by improved clinical symptoms, including lower BP and total cholesterol and weight loss.

“There is a critical need for a medical treatment for people with Cushing's disease because currently available options, such as surgery or radiotherapy, are ultimately not effective for many of the patients who suffer from this debilitating disease,” William H. Ludlam, MD, PhD, Director of the Seattle Pituitary Center at the Swedish Neuroscience Institute, said in a Novartis press release.

Study findings were presented in September at the 14th Congress of the European Neuroendocrine Association in Liege, Belgium.

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