Osilodrostat Phosphate

RECORDATI: FDA GRANTS ISTURISA® (OSILODROSTAT) EXPANDED INDICATION FOR THE TREATMENT OF ENDOGENOUS HYPERCORTISOLEMIA IN PATIENTS WITH CUSHING’S SYNDROME ISTURISA® is a cortisol synthesis inhibitor that blocks the enzyme 11β-hydroxylase to help normalize hypercortisolemia in Cushing’s syndrome, a rare endocrine condition that can have significant impact on patients and their families The expanded indication is supported by an extensive clinical development program April 16, 2025 – Today Recordati announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental new drug application (sNDA) for ISTURISA® (osilodrostat) for the treatment of endogenous hypercortisolemia in adults with Cushing’s syndrome for whom surgery is not an option or has not been curative. This is an expansion of the previous indication for the treatment of patients with Cushing’s disease which is a sub-type of Cushing’s syndrome. The ISTURISA® indication expansion was supported by the ISTURISA® extensive clinical development program which includes over 350 patients. Scott Pescatore, Executive Vice President of Rare Diseases at Recordati, commented, “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. Cushing’s syndrome can often have a devastating impact on the lives of patients and their families. 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 encouraged that more patients are now able to benefit from treatment with ISTURISA® and remain confident in its potential to continue creating important value for the Group, as reflected in the recently updated peak sales expectations for the product.” Maria Fleseriu, MD, FACE, Professor of Medicine and Neurological surgery and director of the Pituitary Center at Oregon Health & Science University and a global PI for LINC studies added, "The expanded indication of osilodrostat 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, this therapy gives me the opportunity to normalize cortisol levels in these patients.” Hypercortisolemia, which is marked by elevated levels of cortisol, is the underlying cause of endogenous Cushing's syndrome, a rare and serious disease of excess cortisol for any reason (pituitary and nonpituitary). Cushing’s disease (a sub-type of Cushing’s syndrome) is cortisol elevated on the basis of pituitary overstimulation (ACTH, adrenocorticotropic hormone) of the adrenal glands. Elevated cortisol can lead to a wide range of associated conditions and complications, such as weight gain, high blood glucose, high blood pressure, osteoporosis, thin and fragile skin that bruises easily, muscle weakness, depression, anxiety, and irritability. If endogenous hypercortisolemia in Cushing’s syndrome is left untreated, it can lead to severe complications and diseases, including diabetes, osteoporosis, cardiovascular issues, and even increased risk of infection due to the suppression of the immune system. ISTURISA® is a cortisol synthesis inhibitor that works by preventing 11β-hydroxylase, an enzyme responsible for the final step of cortisol biosynthesis in the adrenal gland, from being created. ISTURISA® is also approved for the treatment of patients with endogenous Cushing’s syndrome in multiple countries outside the U.S. including the European Union (January 2020) and China (September 2024). ISTURISA® received orphan drug designation from the FDA and the European Medicines Agency for the treatment of endogenous Cushing's syndrome. Recordati is an international pharmaceutical group listed on the Italian Stock Exchange (XMIL: REC), with roots dating back to a family-run pharmacy in Northern Italy in the 1920s. We are uniquely structured to provide treatments across specialty and primary care, and rare diseases. Our fully integrated operations span clinical development, chemical and finished product manufacturing, commercialization and licensing. We operate in approximately 150 countries across EMEA, the Americas and APAC with over 4,450 employees. We believe that health is a fundamental right, not a privilege. Today, our purpose of “unlocking the full potential of life” aims at empowering individuals to live life to the fullest, whether addressing common health challenges or the rarest. The content above comes from the network. if any infringement, please contact us to modify.