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Published on 8 March 2012

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Phase III first for Cushing’s disease drug

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A study published in The New England Journal of Medicine (NEJM) found that the investigational drug Signifor® (SOM230, pasireotide), normalised cortisol levels and showed clinical benefit in patients with Cushing’s disease.

This study, which was first presented at the 14th Congress of the European Neuroendocrine Association in September 2010, is the first Phase III trial to demonstrate the efficacy of a medical therapy for Cushing’s disease.

Cushing’s disease is a debilitating endocrine disorder caused by excess cortisol in the body due to the presence of a non-cancerous pituitary tumor. There are currently no approved medicines that target Cushing’s disease.

In the study, patients were randomised to receive pasireotide subcutaneous (sc) injection in doses of 900µg or 600µg twice daily.

For the 900µg group, the study met the primary endpoint of normalising urinary-free cortisol (UFC) levels, the key measure of biochemical control of the disease.

UFC levels were normalised in 26.3% and 14.6% of patients with Cushing’s disease randomised to receive pasireotide 900µg and 600µg twice daily, respectively, at six months of treatment. After 12 months of treatment, results confirmed the durability of the effect.

“While rare, Cushing’s disease is a serious disease with no cure and very limited treatment options,” said Annamaria Colao, MD, lead study investigator and Professor of Endocrinology, Chief of the Neuroendocrine Unit at the Department of Molecular and Clinical Endocrinology and Oncology, Federico II University of Naples.

“These data on pasireotide are the first to show a therapeutic treatment can help patients achieve biochemical control of their Cushing’s disease, while improving associated symptoms.”

Study results also showed that cortisol levels decreased quickly in the majority of patients, with a median decrease of approximately 50% by month two, and remained stable in both groups through the end of the study.

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

The trial, which represents the largest randomised study to evaluate a medical therapy in patients with Cushing’s disease, is the basis for regulatory submissions for pasireotide under way worldwide for the treatment of this condition.

In January, pasireotide received a positive CHMP opinion for the treatment of Cushing’s disease, and if approved in the EU the brand name will be Signifor.

“These positive study results demonstrate that pasireotide has the potential to be an important therapeutic option for patients living with Cushing’s disease and reinforces Novartis’ commitment to develop therapies to help address unmet medical needs,” said Hervé Hoppenot, President, Novartis Oncology.

“We look forward to working with regulatory authorities worldwide to help bring this novel treatment option to market.”

 



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