The FDA recently approved Corcept Therapeutics’ drug, Korlym (mifepristone), for the treatment of Cushing’s syndrome.
Endogenous Cushing’s syndrome is a rare multisystem disorder caused by overproduction of cortisol, a steroid hormone, which increases blood sugar levels. Korlym was approved for patients with type 2 diabetes or glucose intolerance, that are not considered candidates for surgery, or didn’t respond to surgery in the past.
According to the FDA, Cushing’s syndrome affects people between the age of 25 and 40. Since only about 5,000 patients will be eligible for this drug, the FDA designated Korlym orphan drug status.
A phase III study consisting of 50 patients with endogenous Cushing’s syndrome, who were not eligible or had a relapse from surgery showed that after treatment with Korlym, 60% of patients with glucose intolerant had greater than a 25% improvement in a test.
In the clinical trials, common side effects for patients taking Korlym consisted of nausea, fatigue, headache, arthralgia, vomiting, swelling of the extremities, dizziness and decreased appetite. Korlym should not be taken by women who may be pregnant.
According to Corcept, Korlym will be available in May, at a limited amount of endocrinologists who treat patients with Cushing’s syndrome.