Milan, Italy and Bridgewater, NJ, April 16, 2025 Recordati, the renowned international pharmaceutical group, has announced significant news today, as the U.S. Food and Drug Administration (FDA) granted approval for a supplemental new drug application (sNDA) for ISTURISA (osilodrostat). This newly granted expanded indication allows ISTURISA to be utilized in the treatment of endogenous hypercortisolemia in adults diagnosed with Cushings syndrome, specifically for those patients for whom surgical options are either not viable or have not yielded curative results.

Cushings Syndrome Overview

Cushings syndrome is a rare yet serious endocrine disorder characterized by an overproduction of cortisol, often referred to as the stress hormone. This condition can gravely affect not only the individuals physical health but also their emotional well-being, creating a ripple effect that impacts the lives of their families and loved ones. The new approval marks a critical milestone in providing effective treatment options for patients suffering from this challenging condition.

The expanded indication for ISTURISA is underpinned by a comprehensive clinical development program that included data from over 350 participants. This extensive research highlights the efficacy and safety of the drug, showcasing its potential to alleviate the debilitating symptoms associated with elevated cortisol levels.

Scott Pescatore, Executive Vice President of Rare Diseases at Recordati, expressed his enthusiasm regarding this advancement. He stated, We are pleased that with the label expansion for ISTURISA in the U.S. to address endogenous hypercortisolemia in patients with Cushings syndrome, we can now provide an additional treatment option for an important unmet need. Cushings syndrome can often have a devastating impact on the lives of patients and their families. Elevated cortisol levels, if not properly controlled, can be linked to severe complications such as diabetes, osteoporosis, cardiovascular conditions, and an increased risk of infections. He also noted the updated expectations for peak sales of ISTURISA, reflecting confidence in its market potential.

Dr. Maria Fleseriu, a prominent figure in the field as a Professor of Medicine and Neurological Surgery at Oregon Health & Science University and a global principal investigator for LINC studies, reinforced the significance of this development. She remarked, The expanded indication of osilodrostat is a remarkable progression in the treatment landscape for patients with Cushings syndrome who cannot have surgery or whose condition has not been resolved post-surgery. It allows us the opportunity to normalize cortisol levels in these patients.

Important Safety Information for ISTURISA

ISTURISA is a cortisol synthesis inhibitor designed to manage elevated cortisol levels in the blood of adult patients diagnosed with Cushings syndrome. However, it is crucial to note that the safety and efficacy of ISTURISA in children have not been established.

Patients considering ISTURISA must inform their healthcare provider about their complete medical history. This includes any existing heart conditions, particularly irregular heartbeats or prolonged QT syndrome, as well as any history of low potassium or magnesium levels, liver issues, or pregnancy plans. Women who are breastfeeding should also discuss with their healthcare providers, as it is currently unknown whether ISTURISA can pass into breast milk.

The medication may cause serious side effects, including low cortisol levels, which could lead to symptoms such as nausea, fatigue, low blood pressure, and dizziness. If these occur, patients should seek immediate medical advice. Other possible side effects may include heart rhythm irregularities and hormonal changes, such as increased levels of other adrenal hormones.

ISTURISA is available in tablet form in dosages of 1 mg and 5 mg, and patients are advised to review the full prescribing information before starting treatment.

Understanding Endogenous Hypercortisolemia

Endogenous hypercortisolemia is characterized by excessively high cortisol levels, which can result from various factors, including pituitary tumors that lead to overstimulation of the adrenal glands. This condition is not only linked to physical health issues like weight gain, high blood pressure, and osteoporosis but can also manifest emotionally, causing anxiety and depression.

Without appropriate treatment, patients may face severe complications, including an increased risk of serious infections, which can pose additional health risks. The approval of ISTURISA is a promising development for those affected by this rare condition.

About Recordati

Recordati has a longstanding history in the pharmaceutical industry, with roots tracing back to a family-run pharmacy in Northern Italy in the 1920s. Today, the company operates across approximately 150 countries, with a diverse portfolio that includes treatments for rare diseases. As an organization, Recordati is committed to advancing healthcare and ensuring that essential treatments are accessible to those in need.