Drugs

Guide on Endogenous Cushing’s Syndrome

FDA has published a draft guidance entitled “Endogenous Cushing’s Syndrome: Developing Drugs for Treatment.” The document provides recommendations on clinical trial designs for drugs and biologics intended for treating Cushing’s syndrome in those where surgery is not an option or has not been curative. The agency says the recommendations are not intended for products being developed for treating exogenous Cushing’s syndrome.

FDA says the goal of medical therapy is to control hypercortisolemia either by normalizing cortisol levels (i.e., urinary free cortisol (UFC)) or by blocking the cortisol action at its receptors. “Lifelong medical treatment to suppress cortisol levels and/or action may be required if the primary cause of Cushing’s syndrome cannot be treated successfully with surgery and/or radiation,” it says.

Additionally, the guidance also says that efficacy endpoints should reflect the drug’s mechanism of action. “UFC is a reliable marker to assess the efficacy of drugs that inhibit either pituitary [adrenocorticotropic hormone] ACTH secretion or adrenal steroidogenesis,” it says. “In contrast, UFC is not a reliable biomarker for drugs that block cortisol receptors. Efficacy for this class of drugs should be established by assessing their impact on downstream effects of cortisol at the glucocorticoid receptor.”

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome