The U.S. Food and Drug Administration (FDA) has granted orphan drug status to fluasterone (ST-002) for the treatment of nonalcoholic fatty liver disease, nonalcoholic steatosis, and high blood sugar in Cushing’s syndrome patients, SteroTherapeutics announced.
Cushing’s syndrome occurs when the body is exposed to high levels of the hormone cortisol over a long period. The condition affects between 15,000 and 20,000 people in the United States, according to estimates.
Cortisol is a type of steroid hormone that belongs to the glucocorticoid class of hormones. It has important physiological roles, including regulating blood pressure, limiting the immune system’s response to inflammation, controlling the body’s use of macronutrients, and helping the body respond to stress.
When cortisol levels are too high – a condition known as hypercortisolism – patients can experience harmful physical, mental, and hormonal imbalances.
Too much cortisol can lead to hallmark signs of Cushing’s syndrome, including a fatty hump between the shoulders, a rounded face, and pink or purple stretch marks on the skin. Cushing’s can also result in upper body obesity, diabetes, high bone pressure, and bone loss.
Some of these symptoms are risk factors for nonalcoholic fatty disease and steatosis, two conditions characterized by scarring and irreversible damage to the liver. Because these conditions may cause cirrhosis and consequent liver failure, it is important that Cushing’s syndrome patients receive treatment for nonalcoholic fatty disease and steatosis.
“We are pursuing a drug that has a very real potential to become the optimal agent of choice and a standard of care for these Cushing’s patients,” Manohar Katakam PhD, chief executive officer at SteroTherapeutics, said in a press release. “Our clinical trial will target multiple critical metabolic-related outcomes including the reduction of triglycerides, insulin resistance, weight loss, and the prevention and/or abrogation of hepatic steatosis and fibrosis.”
“The FDA’s orphan-drug designation for Fluasterone highlights the significant unmet and underserved needs for treatment in these individuals,” Katakam added. “We look forward to realizing the benefits and promise of this potential for Fluasterone in Cushing’s syndrome patients.”
The FDA’s orphan drug status is granted to products targeting conditions experienced by fewer than 200,000 people in the United States. The program provides special incentives, including a seven-year period of market exclusivity, tax credits, and assistance with clinical trial design.