On March 06, 2020, the U.S. Food and Drug Administration has announced the approval of new medication Isturisa (osilodrostat), the first and only cortisol synthesis inhibitor and a new non-surgical option for the treatment of patients with Cushing’s Disease (CD)—a rare disease in which patients' adrenal glands produce excessive levels of the hormone cortisol who cannot undergo pituitary gland surgery or have undergone the surgery but still have the disease. (1)
Isturisa (also known as osilodrostat) is a milestone and new non-surgical treatment originally developed by Novartis Pharmaceuticals but acquired by Recordati (an Italy-based European specialty pharmaceutical group) in June 2019 to treat Cushing's Disease. The drugmaker Recordati Rare Diseases Inc. is a public international specialty pharmaceutical company committed to the research and development of new specialties with a focus on treatments for rare diseases. Currently, Recordati has operations throughout the whole of Europe, including the United States of America, Canada, Russia, Turkey, North Africa, Mexico, some South American countries, Japan and Australia.
Recordati Rare Diseases is committed to making this innovative treatment available for all patients suffering from endogenous Cushing’s Syndrome (CS) worldwide and the European approval and subsequent launch is an important first step in this direction according to the drugmaker. (2)
Based on the National Institutes of Health (NIH) study, Cushing Disease is estimated to occur in 10 to 15 per million people worldwide. This disease usually occurs in adults between the ages of 20 and 50; however, children may also be affected. For unknown reasons, Cushing Disease affects females more often than males. (3)
Cushing's Disease is considered a rare condition and caused by elevated levels of a hormone called cortisol. More specifically, this disease is characterized by the hypersecretion of the adrenocorticotropic hormone (ACTH) due to a pituitary adenoma that ultimately causes endogenous hypercortisolism by stimulating the adrenal glands. (4)
Cortisol is a vital hormone that regulates metabolism and cardiovascular function and helps the body respond to stress. Excess levels of cortisol can lead to a wide variety of health issues, such as uncontrollable weight gain, central obesity (abdomen that sticks out with thin arms and legs), a round, red full face, buffalo hump (collection of fat on the back of the neck), type 2 diabetes, blood clotting disorders, thin skin and easy bruising, striae (purple stretch marks) and weak muscles, excess body and facial hair, interrupted menstrual cycle, bone loss and fractures, hypertension (high blood pressure), kidney stones, a weakened immune system and depression. (5) In addition, there is a distinction between Cushing’s Syndrome and Cushing’s Disease. Cushing Disease is a form of Cushing Syndrome. When a pituitary gland tumor secretes excessive amounts of adrenocorticotropic hormone (ACTH), the disorder is referred to as Cushing’s Disease. When the adrenal gland produces excessive amounts of cortisol, the condition is called Cushing’s Syndrome.
The FDA’s approval of Isturisa is based on phase 3 research data generated by the open-label clinical trial, demonstrated that Isturisa inhibits an enzyme called 11-beta-hydroxylase, preventing synthesis of cortisol in 137 adults. Most of those patients had undergone surgery that did not cure their disease or were not candidates for surgery. After 24 weeks, about half of the patients had cortisol levels within a normal range. At that point, 71 patients were entered into an 8-week withdrawal study in which they were randomly assigned to receive either Isturisa or a placebo. At the end of the withdrawal period, the FDA says 86% of patients who received the Novartis drug maintained normal cortisol levels compared to 30% of those who were given a placebo. The results have showed normalization of cortisol levels in a significant portion of adult patients with a manageable safety profile, making this a novel treatment option for patients with Cushing's Disease without surgery. (1)
Isturisa (osilodrostat) is a cortisol synthesis inhibitor indicated for the treatment of adult patients ...
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