FDA Approves New Drug for Cushing’s Disease

The US Food and Drug Administration (FDA) has recently granted a treatment approval for a new drug Isturisa (osilodrostat). The drug can be given to adult patients suffering from a rare type of Cushing’s disease, especially who cannot undergo gland surgery or have undergone surgery but still have persisting condition.

This new drug is now the first FDA approved drug that directly controls the cortisol overproduction by blocking 11-beta-hydroxylase and preventing cortisol synthesis. Previously, there has been no drug that lowers ACTH production or shrinks the pituitary tumor. Adrenocorticotropic hormone (ACTH) is a polypeptide tropic hormone produced by and secreted by the anterior pituitary gland. It is also used as a medication and diagnostic agent. ACTH is an important component of the hypothalamic-pituitary-adrenal axis and is often produced in response to biological stress.

Mary Thanh Hai, M.D., acting director at the Center for Drug Evaluation and Research, FDA, said:

FDA always supports new safe and effective treatment options for rare diseases like Chushing’s disease, in which cortisol overproduction puts patients under high risk. This medicine is now a new and important treatment option for Cushing’s disease and will surely help in maintaining normal cortisol levels in patients.

The approval of Isturisa has been granted to Novartis and will soon hit the market. It will be available in 1 mg, 5 mg and 10 mg film coated oral tablets. It can be taken twice a day as prescribed by health care practitioners.

A study was conducted on 137 adult patients to evaluate the safety and efficacy of this new drug. These patients had undergone surgical pituitary removal but still suffering from disease. After 24 weeks trial period, Isturisa demonstrated normal levels of cortisol in almost 86% patients. The common side effects reported in the study were headache, vomiting, nausea, swelling, adrenal insufficiency and lowering of cortisol levels.

Elevated levels of a steroid hormone (cortisol) in blood lead to a medical condition called Cushing’s syndrome. It is mainly caused by malfunctioning of pituitary gland, leading to an increased secretion of cortisol by adrenal glands. Almost 8 persons in a million in the US are reported with Cushing’s disease each year. The prevalence of this disease is three time more in men between ages 30-50. It can cause health issues like obesity, diabetes, elevated blood pressure, blood clots in lungs and legs, bone abnormalities, depression and weakening of immunity. Abnormalities of muscles and bones may lead to thinning of arms and legs, a red round face and easy bruising.

Source: Research Gate


Novartis is a global Swiss health care concern with a market capital of US $175.6 billion. It manufactures medicines, vaccines, diagnostics and consumer health products. Its aim is to cater patients’ evolving needs by improving quality of life. Some of the famous medicines of the company include Voltaren (diclofenac), clozaril (clozapine), tegretol (carbamazepine), diovan (valsartan), Glivec (imatinib), Ritalin (methylphenidate) and Lamisil (terbinafine). They are used to treat multiple conditions like psychosis, depression, pain and inflammation, gout, epilepsy, high blood pressure, leukemia, narcolepsy and fungal infections.

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