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Endocrinology pipeline

Innovation is at the heart of everything we do, which is why we strive to develop new treatments for rare diseases in endocrinology.

Metyrapone in endogenous Cushing’s syndrome management

Whatever its cause, the clinical consequences of endogenous Cushing’s syndrome are generally severe: glucose tolerance impairment or diabetes, hypertension, dyslipidemia, clotting disorders, vascular fragility, muscular weakness and osteoporosis, diminished resistance to infection, depression and psychiatric disorders, healing defects, gonadal dysfunction with hirsutism, acne. These multiple complications result in significant morbidity and impaired quality of life. Moreover inadequately controlled Cushing’s syndrome is a life-threatening condition, mostly as a result of increased cardiovascular risk.

Metyrapone is an inhibitor of the 11ß-hydroxylase enzyme in the adrenals and thereby blocks cortisol and aldosterone synthesis. 

An international phase 3 / 4 study has been initiated to prospectively confirm metyrapone efficacy and safety in patients with endogenous Cushing’s syndrome.

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