FDA Approves Signifor For Cushing’s Disease

The Food and Drug Administration (FDA) has approved the drug Signifor — the trade name for pasireotide — to treat patients suffering from Cushing’s disease who have failed or are ineligible for surgical treatment.

Cushing’s disease is a condition in which the pituitary gland releases too much adrenocorticotropic hormone (ACTH). ACTH increases the production and release of corticosteroids, which are involved in stress response and regulation of inflammation, among others physiological processes.

The approval of Signifor comes about a month after an advisory committee unanimously endorsed the drug. While there were concerns about hyperglycemia, a condition in which an excessive amount of glucose circulates in the blood plasma, the majority of the panelists agreed that there was a lack of available therapies to treat Cushing’s disease.

Robert Smith, a professor of medicine at Brown University, said:

“In spite of significant concerns about hyperglycemia and uncontrolled diabetes, I think it’s very highly probable that those glucose levels can be adequately controlled with available medications for managing diabetes, including insulin.”

Signifor suppresses the production of cortisol in the adrenal glands, and a clinical trial of 162 patients found that the drug significantly reduces the mean cortisol levels in urine. These findings were the major factor that caused the FDA to approve the drug. The administration did say, however, that patients taking Signifor “need to be carefully monitored for [hyperglycemia] and be treated appropriately with anti-diabetic therapies, including insulin.”

The FDA has recently been criticized for approving drugs — such as Pradaxa — too quickly and without a sufficient review period.

Mary Parks, MD, director of the Division of Metabolism and Endocrinology Products in the FDA’s Center for Evaluation and Research, said in a statement, “Although surgery tends to be first line therapy to treat Cushing’s disease, Signifor is a new treatment option for patients when surgery hasn’t worked or isn’t an option.”

The drug, of course, isn’t with its side effects. They include nausea, abdominal pain, and diarrhea.