FDA Approves New Treatment for Adults with Cushing’s Disease

The US FDA has approved Isturisa (osilodrostat) oral tablets for adults with Cushing’s disease who either cannot undergo pituitary gland surgery or have undergone the surgery but still have the disease. This is the first FDA-approved drug to directly address cortisol overproduction by blocking the enzyme known as 11-beta-hydroxylase and preventing cortisol synthesis.

Osilodrostat had previously received Orphan Drug Designation.

“The FDA supports the development of safe and effective treatments for rare diseases, and this new therapy can help people with Cushing’s 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 said in a press release. “By helping patients achieve normal cortisol levels, this medication is an important treatment option for adults with Cushing’s disease.”