FDA Grants ST-002 Orphan Drug Designation for Cushing


The FDA granted orphan drug designation to fluasterone (ST-002) for the treatment of Cushing’s syndrome.

This week, the U.S. Food and Drug Administration (FDA) granted SteroTherapeutics orphan drug designation for fluasterone (ST-002), its product intended for the treatment of nonalcoholic fatty liver disease, nonalcoholic steatosis, and hyperglycemia in patients with Cushing’s syndrome.

Cushing’s syndrome occurs when a patient’s body is exposed to high levels of the hormone cortisol over long periods of time (chronic hypercortisolemia). Cushing syndrome, oftentimes called hypercortisolism, affects 15,000 to 20,000 patients in the United States.

Fluasterone (ST-002) is a synthetic steroid that has numerous potential therapeutic and preventive uses. In Phase 1 clinical trials, the drug was shown to be safe and well tolerated at high doses while not being metabolized to testosterone or estrogen in men or women.

Manohar Katakam, PhD, CEO of SteroTherapeutics, stated his anticipation for the drug’s potential. “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. 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.”1

“The FDA’s orphan-drug designation for Fluasterone highlights the significant unmet and underserved needs for treatment in these individuals,” added Katakam. “We look forward to realizing the benefits and promise of this potential for Fluasterone in Cushing’s syndrome patients.”

The FDA’s orphan drug designation provides a 7-year period of U.S. marketing exclusivity upon regulatory approval of the drug, as well as tax credits for clinical research costs, annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

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