FDA approves first Cushing's syndrome drug, Corcept's Korlym

Shares of USA-based Corcept Therapeutics (Nasdaq: CORT) leapt 62% to $4.90 in after-hours trading on Friday, when the US Food and Drug Administration approved that company’s Korlym (mifepristone) for the control of hyperglycemia (high blood sugar levels) in adults with endogenous Cushing’s syndrome.

The drug was approved 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. However, although pregnancy is an extremely rare occurrence in Cushing’s syndrome patients because of the suppressive effect of excess cortisol on female reproductive function, Korlym will carry a Boxed Warning advising health care professionals and patients that the therapy will terminate a pregnancy. The active ingredient of Korlym, mifepristone, is also used in the abortion pill RU-486.

Joseph Belanoff, the company's chief executive, said "We plan to make Korlym available to patients by May 1 through a distribution system designed to support both patients and prescribers." The company has voluntarily proposed distributing Korlym through a central pharmacy to ensure the timely, convenient and appropriate delivery of the drug to Cushing’s patients or to the health care institutions where this therapy may be initiated. Most retail pharmacies are unlikely to keep adequate supplies of the drug for this rare condition and central distribution will give patients with Cushing’s syndrome better access to Korlym.