MENLO PARK, CA—Corcept Therapeutics Inc., a companyspecializing in the development of diseases caused by excess or irregularcortisol levels, has announced that the U.S. Food and Drug Administration hasgranted approval for Korlym as a once-daily oral medicine to controlhyperglycemia secondary to hypercotisolism in adult patients with endogenousCushing's syndrome who have diabetes mellitus type 2 or glucose intolerance andhave failed surgery or are not appropriate candidates for surgery. Corcept willbe the sole marketer of the medicine, with CuraScript SP, a subsidiary ofExpress Scripts, dispensing the drug.
"Korlym is a significant advance in the treatment ofpatients suffering from the debilitating symptoms of Cushing's syndrome,"Robert L. Roe, M.D., Corcept's President, said in a press release. "Forthe first time, these patients have access to an approved therapy when surgeryhas failed or is not an option."
"We appreciate the FDA's diligent attention to our NDAand its grant of approval on the PDUFA date," Joseph K. Belanoff, M.D.,Chief Executive Officer of Corcept, said in a press release. "We plan tomake Korlym available to patients by May 1 through a distribution systemdesigned to support both patients and prescribers."
Endogenous Cushing's syndrome is a rare and potentiallyfatal endocrine disorder resulting from long-term exposure to excess levels ofcortisol, caused by tumors in the pituitary or adrenal glands. In excess,cortisol causes hyperglycemia, stretch marks, upper body obesity, thin andeasily bruised skin, muscle and bone weakness, infections, high blood pressure,fatigue, anxiety, psychosis and depression, to name a few symptoms. More than70 percent of patients with Cushing's syndrome suffer from glucose intoleranceor diabetes. Treatment depends on the cause, and can either consist of surgeryto remove the tumor or radiation, though the latter can take up to 10 years fora full effect. Both options are only successful in approximately 50 percent ofall cases, and if untreated, patients with Cushing's syndrome have a five-yearmortality rate of 50 percent.
Korlym's approval was based on an uncontrolled, open-label,multi-center, 24-week Phase III study of 50 patients with endogenous Cushing'ssyndrome who were either ineligible for or had relapsed from surgery and wereeither glucose intolerant or suffering hypertension. Of the glucose intolerantgroup, 60 percent had a greater than 25 percent reduction from baseline in thearea under the curve in the oral glucose tolerance test, and mean hemoglobinA1C was reduced from 7.4 percent to 6.3 percent. All 14 patients exhibitingabove-average HbA1C levels at baseline saw reductions, and eight of themnormalized their HbA1C. Antidiabetic medications were reduced in seven of the15 with diabetes mellitus type 2 and stayed constant in the others.
"There are not many effective treatment options forpatients with Cushing's syndrome. Although surgery is standard first linetreatment for the disease, it is not always successful and not all patients arecandidates," Amir Hamrahian, M.D., Department of Endocrinology, Diabetes andMetabolism at the Cleveland Clinic, said in a press release. Hamrahian was theKorlym clinical trial investigator. "As part of the clinical trial, I have usedKorlym successfully and my patients continue to do well on the medicine. I'mexcited to be able to continue using Korlym in these patients and others whoneed it. This medicine's approval gives me a much needed tool to better treatpatients."
SOURCE: Corcept press release