RECORDATI: FDA GRANTS ISTURISA® (OSILODROSTAT) EXPANDED INDICATION FOR THE TREATMENT OF ENDOGENOUS HYPERCORTISOLEMIA IN PATIENTS WITH CUSHING’S SYNDROME

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 11ß-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

Milan, Italy and Bridgewater, NJ, 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.”

Consumer Important Safety Information

WHAT is ISTURISA?

ISTURISA is a prescription medicine used to treat elevated levels of cortisol in the blood (endogenous hypercortisolemia) in adults with Cushing’s syndrome:

• who cannot have surgery, or
• who have had surgery which did not cure their Cushing’s syndrome

It is not known if ISTURISA is safe and effective in children.

IMPORTANT SAFETY INFORMATION

Before starting ISTURISA tell your healthcare provider about all your medical conditions, including if you:

• have or had heart problems, such as an irregular heartbeat, including a condition called prolonged QT syndrome (QT internal prolongation). Your healthcare provider will check the electrical signal of your heart (called an electrocardiogram) before you start taking ISTURISA, 1 week after starting ISTURISA, and as needed after that.
• have a history of low levels of potassium or magnesium in your blood.
• have liver problems.
• are or plan to become pregnant. It is not known if ISTURISA will harm your unborn baby. There are risks to the mother and unborn baby associated with active Cushing’s syndrome during pregnancy.
• are breastfeeding or plan to breastfeed. It is not known if ISTURISA passes into your breast milk. You should not breastfeed if you take ISTURISA and for 1 week after stopping treatment.
  
  

Tell your healthcare provider about all the medicines you take, including any prescription and over-the-counter medicines, vitamins, or herbal supplements.

Especially tell your healthcare provider if you take medicines used to treat certain heart problems. Ask your healthcare provider if you are not sure whether your medicine is used to treat heart problems.

ISTURISA can cause serious side effects including:

• Low cortisol levels in your blood (hypocortisolism). Tell your healthcare provider right away if you experience more than one of the following symptoms, as these may be symptoms of very low cortisol level, known as adrenal insufficiency: nausea, vomiting, tiredness (fatigue), low blood pressure, problems with body salt (electrolyte) levels in your blood, stomach (abdominal) pain, loss of appetite, dizziness, low blood sugar.
  
  If you get symptoms of hypocortisolism while taking ISTURISA, your healthcare provider may change your dose or ask you to stop taking it.
• Heart problem or a heart rhythm problem, such as an irregular heartbeat which could be a sign of a heart problem called QT prolongation. Call your healthcare provider right away if you have irregular heartbeats.
• Increase in other adrenal hormone levels. Your other adrenal hormones may increase when you take ISTURISA. Your healthcare provider may monitor you for the symptoms associated with these hormonal changes while you are taking ISTURISA:
  • Low potassium (hypokalemia).
  • High blood pressure (hypertension).         
  • Swelling (edema) in the legs, ankles or other signs of fluid retention.
  • Excessive facial or body hair growth (hirsutism).
  • Acne (in women).

Call your healthcare provider if you have any of these side effects.

The most common side effects of ISTURISA include very low cortisol levels (adrenal insufficiency), tiredness (fatigue), nausea, headache, and swelling of the legs, ankles or other signs of fluid retention (edema).

These are not all the possible side effects of ISTURISA. Call your healthcare provider for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Call 1-800-FDA-1088 or visit www.fda.gov/medwatch.

ISTURISA^® (osilodrostat) tablets, for oral use, is available as 1 mg and 5 mg tablets

Please see full Prescribing Information

About Endogenous Hypercortisolemia in Cushing’s Syndrome

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.

About ISTURISA^®
ISTURISA^® is a cortisol synthesis inhibitor that works by preventing 11ß-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.  

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