FDA expands Isturisa approval to all forms of endogenous Cushing’s
Therapy now OK’d for adults when surgery isn't option or wasn’t curative
The U.S. Food and Drug Administration (FDA) has approved Isturisa (osilodrostat) to treat adults with all forms of endogenous Cushing’s syndrome for whom surgery is not an option or has not been curative.
This marks an expansion of the approved indication for Isturisa, which had previously been cleared only for use by adults with Cushing’s disease, a specific subtype of Cushing’s syndrome.
The therapy is marketed by Recordati Rare Diseases, which noted in a company press release that “the expanded indication is supported by an extensive clinical development program.”
“We are encouraged that more patients are now able to benefit from treatment with Isturisa,” said Scott Pescatore, Recordati’s executive vice president.
Isturisa first approved in US in 2020 only for Cushing’s disease
Cushing’s syndrome is a broad term for health conditions driven by hypercortisolemia, or abnormally high levels of the hormone cortisol. In endogenous Cushing’s syndrome, high cortisol levels are caused by problems within the body itself, usually a tumor. Cushing’s disease is the most common form of endogenous Cushing’s syndrome; it is specifically caused by a tumor that forms in the brain’s pituitary gland.
In all forms of Cushing’s syndrome, excess cortisol can lead to a wide variety of symptoms, which include weight gain, unstable mood, fatigue, skin changes, and muscle weakness.
“Cushing’s syndrome can often have a devastating impact on the lives of patients and their families,” Pescatore said. “Elevated cortisol levels in Cushing’s syndrome, if not properly controlled, can be associated with severe complications such as diabetes, osteoporosis, cardiovascular and increased risk of infections.”
We are pleased that with the label expansion for Isturisa in the U.S. to endogenous hypercortisolemia in patients with Cushing’s syndrome, this important unmet need can now be addressed with a further treatment modality.
Isturisa is a cortisol synthesis inhibitor that works by blocking the activity of 11-beta-hydroxylase, an enzyme involved in cortisol production. The therapy was originally approved in the U.S. in 2020 as a treatment for Cushing’s disease.
“We are pleased that with the label expansion for Isturisa in the U.S. to endogenous hypercortisolemia in patients with Cushing’s syndrome, this important unmet need can now be addressed with a further treatment modality,” Pescatore said.
The FDA’s decision to expand the approval of Isturisa to cover all forms of endogenous Cushing’s syndrome was based on data from a suite of clinical trials. Collectively, these studies tested the therapy in more than 350 patients.
LINC-3 (NCT02180217) and LINC-4 (NCT02697734) were Phase 3 trials that specifically tested Isturisa in people with Cushing’s disease. LINC-6 (NCT05382156) and LINC-7 (NCT05633953) enrolled individuals with other forms of endogenous Cushing’s syndrome.
Maria Fleseriu, MD, a global principal investigator for the LINC studies at Oregon Health and Science University, said the expanded approval of Isturisa “is a significant advancement in the treatment of patients with Cushing’s syndrome for whom surgery is not an option or has not been curative.”
Fleseriu said the FDA expansion of the therapy’s approval “gives me the opportunity to normalize cortisol levels in these patients.”
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