Amylin submits metreleptin rolling submission BLA for treatment of rare forms of lipodystrophy

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Amylin Pharmaceuticals, Inc. (Nasdaq: AMLN) today announced that it has submitted the initial sections of a rolling submission for a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the use of metreleptin to treat diabetes and/or hypertriglyceridemia (high levels of triglycerides in the bloodstream) in patients with rare forms of lipodystrophy. Consistent with the severity and rare nature of the disorder, Amylin has received both orphan drug designation from FDA's Office of Orphan Products Development, as well as Fast Track designation for the use of metreleptin in patients with lipodystrophy. The focus of this marketing application is on rare inherited and acquired forms of lipodystrophy.

In the first part of its rolling submission, the Company submitted the nonclinical and clinical sections of the BLA. The Company plans to submit the chemistry, manufacturing and controls (CMC) section of the BLA by the end of 2011, which will complete the submission.

"It is gratifying to see that, after years of research focused on leptin as an effective therapy for lipodystrophy, we are now closer to bringing this important and innovative medicine to patients who are in dire need of better treatments," said Phillip Gorden, M.D., Director Emeritus, Senior Investigator, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at the National Institutes of Health (NIH). Dr. Gorden is the principal investigator of an ongoing NIH clinical study evaluating the long-term efficacy of metreleptin treatment in lipodystrophy.

Lipodystrophy syndromes are characterized by abnormalities in adipose (fat) tissue distribution with loss of subcutaneous fat, and often manifest in childhood or adolescence. Patients with lipodystrophy can have multiple severe metabolic abnormalities, including extreme insulin resistance, very high triglyceride levels, difficult-to-control diabetes and hepatic steatosis (excess fat accumulation in the liver). These abnormalities result in a high risk for serious medical problems such as acute pancreatitis, accelerated atherosclerosis, vessel and nerve damage from diabetes and liver cirrhosis, which can markedly reduce quality of life and life expectancy. Because patients with lipodystrophy do not have enough fat tissue, they typically also have a deficiency of leptin, a hormone secreted by fat cells that plays a key role in regulating metabolism. Metreleptin therapy, an analog of the human hormone leptin, can substantially reduce high glucose and triglyceride levels in these patients. If approved, metreleptin would be the first therapy indicated specifically for the treatment of diabetes and high triglycerides in patients with lipodystrophy, and the first approved therapeutic use of leptin.

"We are proud to advance metreleptin to this regulatory milestone, and look forward to working with the FDA to make this important treatment more broadly available to patients with lipodystrophy," said Daniel M. Bradbury, President and Chief Executive Officer at Amylin. "The use of metreleptin to treat this rare and debilitating condition represents another example of the tremendous potential for peptide and protein science to be translated into therapies that improve the lives of patients with metabolic disorders."

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