"This type of therapy provides an alternative for patients who have not responded to other therapies," said Steven Galson, M.D., M.P.H., director, Center for Drug Evaluation and Research. "The new approval reflects FDA's goals for making effective and safe treatments available to patients with rare diseases under the Orphan Drugs program."

FDA has approved Somatuline Depot for the long-term treatment of patients with acromegaly who have had inadequate response to or can not be treated with surgery and/or radiation therapy. This new treatment lowers the levels of certain hormones in the body, including GH and insulin-like growth factor. Excessive GH secretion, working through insulin-like growth factor, can cause enlargement of the hands, feet, facial bones, and enlargement of internal organs such as the heart and liver. If untreated, patients with acromegaly often have a shortened life span because of heart and respiratory diseases, diabetes mellitus, and colon cancer.

The safety and effectiveness of Somatuline Depot (administered through injection) was determined in two pivotal clinical trials involving a total of 400 patients. Common side effects include diarrhea, gallstones, skin reactions such as itching, slow heart rate, and change in blood sugar levels. Patients with diabetes who receive treatment with Somatuline Depot may need to have their diabetes medication adjusted.

FDA designated Somatuline Depot orphan status because the drug treats a rare disease and meets other criteria. Orphan products are developed to treat rare diseases or conditions that affect fewer than 200,000 people in the United States. The Orphan Drug Act provides a seven-year period of exclusive marketing to the first manufacturer who obtains marketing approval for a designated orphan product. Acromegaly affects approximately 15,000 people in the United States and Canada and is most commonly found in middle-aged adults. Patients with acromegaly have reduction in life expectancy of 5 to 10 years.

The drug will be marketed by Beaufour Ipsen, Paris, France.

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