From Dr. Theodore Friedman: Cushings-Help.com Announces: The Availability Of An Investigational Drug For Severe Cushing’s Syndrome On a Compassionate Use Basis
We would like to make patients aware that mifepristone, an investigational drug that blocks the action of cortisol and is being developed by Corcept Therapeutics Incorporated, is now available on a compassionate use basis for eligible patients in the United States with Cushing’s syndrome who have no other treatment options.
Under this compassionate use program, the FDA allows seriously ill patients who lack satisfactory alternative treatment options to use an investigational new drug that is still under development. Corcept has completed a Phase III trial investigating the safety and efficacy of mifepristone in patients with endogenous Cushing’s syndrome. The information from that study has been submitted to the FDA for review of safety and efficacy.. For information on the trial results see http://www.corcept.com/cushings_clinical_trials. The company has submitted a New Drug Application (NDA) seeking approval for this drug.
Patients interested in using mifepristone should consult with their endocrinologist. Their endocrinologist, in turn, should contact Corcept for information about the compassionate use program. Please note that Corcept will provide information solely to physicians.
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Corcept Therapeutics: Clinical Development and Trials