As originally reported in Biospace, Cushing disease, a disease due to excessive cortisol, has recently had its first ever treatment option approved by the FDA for adult patients who are unable to have the available surgery options. The drug, Isturisa (osilodrostat), is owned by the Italian firm Recordati after they acquired it in summer of 2019. It is the first drug to prevent a particular enzyme from synthesizing cortisol, which the disease causes to be produced in too high a quantity.
Cushing disease is a rare disease caused by excessive production by the pituitary gland, initiated by a benign tumor or excessive growth. This results in too much of the adrenocorticotropic hormone (ACTH) and cortisol. Cortisol is known as the stress hormone because it is triggered by it, causing higher blood pressure and reducing inflammation, speeding up the production and breakdown of proteins, carbohydrates, and fats. However, with proper functioning, this maintains healthy cell metabolization. With too much of it, it causes Cushing disease. Symptoms are diverse among patients, but they it often begins with weight gain around the neck and face and in time can cause higher blood pressure, a weak immune system, type 2 diabetes, mood changes, depression, anxiety, fragile bones, fatigue, and changes in reproductive functions. The disease is much more common in women, who make up about 75% of the patients. Most patients are middle aged, being diagnosed between ages 20 and 50.
The New Treatment Option
The new drug is intended for patients who either had unsuccessful pituitary surgery or could not have it. The phase III trial sampled 137 adults who were given Isturisa and a control group with a placebo. After six months, half of the patients taking the drug had normal cortisol levels. The patients who did not need drug increases at this point were then given another trial that either gave them more of the drug or the placebo, at which point 86% of those on the drug remained healthy. Following these successful trial results, the drug has been approved by both the European Commission and FDA.