The FDA has approved Korlym (mifepristone; Corcept Therapeutics), a once-daily oral medication, for use in patients with endogenous Cushing’s syndrome who have type 2 diabetes or glucose intolerance and are not candidates for surgery or who have not responded to prior surgery. Endogenous Cushing’s syndrome is a serious, debilitating, and rare multisystem disorder. This syndrome most commonly affects adults between the ages of 25 and 40. About 5,000 patients will be eligible for Korlym treatment, which received an orphan drug designation by the FDA in 2007.

Endogenous Cushing’s syndrome is caused by the overproduction of cortisol by the adrenal glands. Korlym works by blocking glucocorticoid receptor type II to which cortisol normally binds. It does not decrease cortisol production but reduces the effects of excess cortisol, such as high blood-sugar levels.

The safety and efficacy of Korlym in patients with endogenous Cushing’s syndrome was evaluated in a clinical trial with 50 patients. A separate open-label extension of this trial is ongoing. Additional evidence supporting the FDA’s approval included several safety pharmacology studies, drug-drug interaction studies and published scientific literature. Patients experienced significant improvement in blood-sugar control during Korlym treatment, including some patients who had marked reductions in their insulin requirements. Improvements in clinical signs and symptoms were reported by some patients.

Korlym is expected to be available in a 300mg tablet dosage form by May 1, 2012.

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