HealthDay News — The FDA has approved mifepristone (Korlym, Corcept Therapeutics) to treat endogenous Cushing’s syndrome in patients with type 2 diabetes who have not responded to previous surgery or are not candidates for new surgery, the agency announced in a press release.
Mifepristone is the first approved medical therapy for this debilitating, albeit rare, multisystem disorder. The agency projects that only about 5,000 people in the United States are likely to be eligible for the drug.
Data from clinical trials involving 50 patients showed that those taking mifepristone experienced significant improvements in blood glucose control compared to patients who did not take the medication, and some patients taking mifepristone reported improvements in clinical signs and symptoms of the disorder. A separate open-label extension of this trial is ongoing.
Mifepristone reduces the effects of cortisol overproduction, rather than decreasing production, according to the FDA. Common side effects observed during clinical testing included nausea and vomiting, fatigue, headache, swelling of the extremities, dizziness and loss of appetite.
Clinicians should also be aware that combining mifepristone with other drugs could increase drug concentrations and should take care to adjust dosing or avoid using certain drugs with mifepristone, the FDA warned, adding that a detailed medication guide will be available for clinicians and patients.
Although pregnancy is an extremely rare occurrence in Cushing’s syndrome, mifepristone will carry a boxed warning indicating that expecting women should never use the drug as it is likely to induce termination.