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Published on 22 September 2010

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SOM230 is first medical therapy to show efficacy in a Phase III trial in Cushing’s disease

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Novartis announced today that the results of a Phase III study of SOM230 (pasireotide) showed a reduction in cortisol levels in patients with Cushing’s disease, a condition in which a benign (non-cancerous) pituitary tumor causes the adrenal glands to produce excess cortisol and can be fatal. Results will be presented at the 14th Congress of the European Neuroendocrine Association (ENEA).

At six months, the majority of evaluable patients (91/103) experienced a reduction from baseline in urinary free cortisol (UFC) levels, the main measure of biochemical control of the disease. UFC levels were normalized in 26% of patients randomised to SOM230 900µg twice daily, meeting the primary endpoint of the study. Additionally, median UFC was reduced by 48% in both the 900µg and 600µg dose groups. After 12 months of treatment, results confirmed the durability of the effect.

On average, as UFC levels were reduced, clinical symptoms of Cushing’s disease improved including reduction of blood pressure, total cholesterol, weight and body mass index (BMI).

Cushing’s disease is caused by a benign tumor in the pituitary gland that secretes adrenocorticotropic hormone (ACTH), which triggers the adrenal glands to produce excess cortisol. Cortisol is a powerful steroid hormone that regulates a broad range of physiologic functions, including metabolism and immunity. Cushing’s disease can cause severe cardiovascular and metabolic-related illnesses or death. There are currently no approved medicines to treat Cushing’s disease.

“Up to half of patients with Cushing’s disease cannot be cured with currently available options, which include surgery or radiotherapy of the tumor, leaving a critical need for medical treatments,” said Annamaria Colao, MD, Professor of Endocrinology and Chief of the Neuroendocrine Unit at the Department of Molecular and Clinical Endocrinology and Oncology, Federico II University of Naples and one of the study investigators. “The study findings show that SOM230 has the potential to directly target the underlying pituitary tumor and suppress cortisol production, thereby helping patients achieve biochemical control of their Cushing’s disease and the associated debilitating symptoms.”

PASPORT-CUSHINGS (PASireotide clinical trial PORTfolio – CUSHING’S disease) is the largest randomised study to evaluate a medical therapy in patients with Cushing’s disease. The trial is part of a large-scale global clinical development program to assess the efficacy and safety of SOM230 in a range of pituitary and neuroendocrine tumors.

“Positive results from this trial bring us one step closer to providing physicians with a new treatment option to offer people living with the physically and emotionally debilitating symptoms associated with Cushing’s disease,” said Hervé Hoppenot, President, Novartis Oncology.

These data will form the basis for the first regulatory filing of SOM230 planned this year. SOM230 has orphan drug designation for Cushing’s disease in the US and Europe. In the US, orphan drugs are those developed to treat diseases affecting fewer than 200,000 people. In Europe, orphan drugs are those developed to treat conditions affecting fewer than five in 10,000 people.

Novartis



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