FDA Approves New Drug for Cushing Disease

The US Food and Drug Administration (FDA) has approved Isturisa (osilodrostat) oral tablets for adults with Cushing disease who are either unable to undergo pituitary gland surgery or have undergone the surgery but still have the disease.

Isturia is the first FDA-approved drug to address the overproduction of cortisol in Cushing disease by blocking the enzyme known as 11-beta-hydroxylase and preventing cortisol synthesis, according to the FDA. The drug is to be taken orally twice daily (once in the morning and evening). After treatment has started, health care providers may reevaluate dosage, depending upon the patient’s response.

The most commonly reported adverse effects in the clinical trial for Isturisa were adrenal insufficiency, headache, vomiting, nausea, fatigue and edema. Hypocortisolism, QTc prolongation, and elevations in adrenal hormone precursors and androgens may also occur in patients being treated with Isturisa.

“The FDA supports the development of safe and effective treatments for rare diseases, and this new therapy can help people with Cushing disease, a rare condition where excessive cortisol production puts them at risk for other medical issues,” said Mary Thanh Hai, MD, acting director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research, in a press release.

“By helping patients achieve normal cortisol levels, this medication is an important treatment option for adults with Cushing disease,” said Dr Thanh Hai in a press release.

—Christina Vogt

FDA approves new treatment for adults with Cushing’s disease. News release. US Food and Drug Administration. March 6, 2020.