Pipeline / Endocrine Diseases

Mifedren^®

Mifedren^® (HRA052015) is our product candidate for a subset of Cushing's syndromes that is caused by ectopic ACTH* secretion. Cushing's syndrome is a hormonal disorder caused by prolonged exposure of the body's tissues to the hormone cortisol. The condition is characterized by upper body obesity, osteoporosis, hypertension, diabetes mellitus, hirsutism, amenorrhea and excess body fluid. Cushing's syndrome has several causes depending on the source of the cortisol that is secreted in excess. Some Cushing's syndromes are linked to the abnormal (ectopic) secretion of another hormone, ACTH, from a specific kind of tumor. Mifedren^® shows promise for the treatment of the symptoms of this particular subset of the disease. Around three in every 1,000,000 people are diagnosed with Cushing's syndrome due to ectopic ACTH secretion each year in the USA and in Europe. Mifedren^® is currently in Phase II/III trials for this disease. Given the size of the population that is affected by this rare disease, Mifedren^® has been awarded orphan-drug status in both Europe and the United States.

*ACTH: Adrenocorticotropic Hormone