FDA Approves ISTURISA® (Osilodrostat) for Expanded Use in Cushing’s Syndrome

In a significant advancement for patients suffering from Cushing's syndrome, the FDA has granted an expanded indication for ISTURISA® (osilodrostat) to treat endogenous hypercortisolemia. This approval opens new doors for adults who cannot undergo surgery or whose surgery has not resolved their condition.

What is ISTURISA®?

ISTURISA® is a prescription medication designed to:

• Inhibit cortisol synthesis: It blocks the enzyme 11β-hydroxylase, which is essential for cortisol production in the adrenal glands.
• Normalize cortisol levels: By reducing excessive cortisol, it helps manage symptoms associated with Cushing’s syndrome.

Key Points about Cushing's Syndrome

Cushing's syndrome is a rare endocrine disorder characterized by elevated cortisol levels. This condition can lead to serious health complications, including:

• Weight gain
• High blood pressure
• High blood glucose levels
• Osteoporosis
• Mood changes, including anxiety and depression

If left untreated, high cortisol levels can result in severe health issues, such as diabetes, cardiovascular problems, and increased susceptibility to infections.

Expanded Indication Details

The FDA's approval for ISTURISA® is backed by a comprehensive clinical development program that included over 350 patients.

Comments from Experts

• Scott Pescatore, Executive Vice President of Rare Diseases at Recordati, expressed the importance of this approval, stating, "This important unmet need can now be addressed with a further treatment modality."

• Dr. Maria Fleseriu, a leading expert in the field, highlighted the significance of this therapy, stating, "This therapy gives me the opportunity to normalize cortisol levels in these patients."

Important Safety Information

Who Should Use ISTURISA®?

ISTURISA® is intended for adults with Cushing’s syndrome who:

• Cannot have surgery
• Have undergone surgery that was not curative

Potential Side Effects

ISTURISA® may cause serious side effects, including:

• Low cortisol levels (hypocortisolism): Symptoms may include nausea, fatigue, low blood pressure, and dizziness.
• Heart rhythm issues: Irregular heartbeats can indicate potential heart problems.
• Hormonal changes: Increased levels of other adrenal hormones may lead to symptoms such as low potassium, high blood pressure, and excessive hair growth.

Common Side Effects

• Adrenal insufficiency (very low cortisol levels)
• Fatigue
• Nausea
• Headache
• Edema (swelling in the legs or ankles)

Important Precautions

Before starting ISTURISA®, inform your healthcare provider if you have:

• A history of heart problems, low potassium, or liver issues
• Plans to become pregnant or are breastfeeding

Conclusion

The FDA's expanded indication for ISTURISA® marks a pivotal moment in the treatment of Cushing’s syndrome, offering new hope to those who face challenges in managing their condition. With this approval, patients can access a vital therapy that may enhance their quality of life and mitigate the severe effects of elevated cortisol levels.

Disclaimer: This article is for informational purposes only and does not constitute financial advice. Always do your own research or consult a licensed financial advisor before making investment decisions.