Recordati Rare Diseases, Inc. announced the availability of Isturisa® (osilodrostat) tablets for the treatment of adults with Cushing disease for whom pituitary surgery is not an option or has not been curative.

Isturisa is a cortisol synthesis inhibitor that works by blocking the enzyme 11-beta-hydroxylase, which is responsible for the final step of cortisol biosynthesis in the adrenal gland. The approval was based on data from a pivotal phase 3 study (N=137), which included a 24-week, single-arm, open-label titration and treatment period and an 8-week, double-blind, randomized withdrawal period. Results showed that Isturisa met the primary end point with a significantly greater proportion of patients maintaining normal mean urinary free cortisol (mUFC) at the end of the 8-week randomized withdrawal period (week 34) vs patients switched to placebo (86% vs 29%). 

With regard to safety, the most common adverse reactions reported were adrenal insufficiency, fatigue, nausea, headache, and edema. Treatment with Isturisa has also been associated with an increased risk of hypocortisolism, QTc prolongation, and elevations in adrenal hormone precursors and androgens. 

Prior to initiating treatment, hypokalemia and hypomagnesemia should be corrected. In addition, an electrocardiogram should be obtained at baseline and repeated within 1 week after initiation, and as clinically indicated thereafter.

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“Isturisa mechanistically inhibits the final step of the cortisol synthesis pathway,” said Richard J. Auchus MD, PhD, a professor of medicine at the University of Michigan School of Medicine. “Achieving control of cortisol production is an important strategy to help patients manage Cushing disease, and it may be crucial for mitigating the risk for comorbidities associated with hypercortisolism.”

Isturisa is exclusively available through the R.A.R.E.™ (Recordati, Access, Resources, and Engagement) Patient Support Program and distributed via AnovoRx specialty pharmacy. The program offers financial assistance among other services to eligible patients to help gain access to the treatment.

The product is supplied as 1mg, 5mg, and 10mg strength tablets in cartons of three 20-count blister packs. 

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