Amylin Completes Biologics License Application for Metreleptin to Treat Diabetes and/or Hypertriglyceridemia in Patients With Rare Forms of Lipodystrophy

If Approved, Metreleptin Would Represent First Therapy for Life-Threatening Metabolic Disease

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Apr 03, 2012, 08:00 ET

SAN DIEGO, April 3, 2012 /PRNewswire/ -- Amylin Pharmaceuticals, Inc. (Nasdaq: AMLN) today announced that it has completed its Biologics License Application (BLA) submission 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 pediatric and adult patients with rare forms of lipodystrophy.

The rolling BLA submission was initiated in December 2010 with the clinical and nonclinical sections; Amylin has now submitted the chemistry, manufacturing, and controls (CMC) section to complete the BLA. Metreleptin has Fast Track designation for use in lipodystrophy patients.

Amylin also requested Priority Review, a designation given to drug candidates that offer major advances in treatment, or provide a treatment where no adequate therapy exists. The FDA typically allocates additional resources to review an application that has been designated Priority Review status under a shortened timeline.

Lipodystrophy is a life-threatening, "ultra orphan" rare disease that is estimated to impact a few thousand people worldwide, often with an early age of onset, and represents a significant unmet medical need as there are no approved drugs that treat the underlying cause of the disease.

"Completion of this submission is an important milestone for Amylin and for patients with rare forms of lipodystrophy who currently have limited, and often insufficient, treatment options for this under-recognized and life-threatening disease," said Daniel M. Bradbury, president and chief executive officer of Amylin. "Metreleptin is an integral component of our ongoing commitment to improve the lives of patients with metabolic diseases – from the rarest to the most prevalent."

Fat tissue is a major endocrine organ producing important metabolic hormones such as leptin. People with lipodystrophy lack the required fat tissue for normal metabolic function. This can be partial, affecting select areas of the body, or generalized, affecting nearly the entire body. A lack of fat tissue can lead to relative deficiency of leptin. Without adequate leptin function, the metabolic system, which regulates food intake and the storage and break-down of dietary fat and carbohydrates, falls out of balance. As a result, fat accumulates in the blood and organs such as liver and muscle, which can lead to life-threatening complications including insulin-resistant diabetes, hypertriglyceridemia, acute pancreatitis, and hepatic steatosis or steatohepatitis, also known as fatty liver disease. There are no approved drugs that address the underlying relative leptin deficiency that is believed to contribute in large part to the metabolic abnormalities that occur in lipodystrophy. Currently available therapies for diabetes and hypertriglyceridemia are often rendered marginally effective due to the severity of the condition.

About Metreleptin

Metreleptin, an analog of the human hormone leptin, is a unique potential therapy for patients with lipodystrophy because it addresses the relative leptin deficiency in this disease. Its mechanism of action includes reducing fat accumulation in organs thus improving insulin sensitivity. In clinical studies conducted by investigators at the National Institutes of Health (NIH) and other academic institutions in the U.S., Europe, and Japan, metreleptin has been shown to improve glycemic control and hypertriglyceridemia in patients with lipodystrophy.

About Amylin Pharmaceuticals

Amylin Pharmaceuticals is a biopharmaceutical company dedicated to improving lives of patients through the discovery, development, and commercialization of innovative medicines. Amylin is committed to delivering novel therapies that transform the way diabetes and other metabolic disorders are treated. Amylin is headquartered in San Diego, Calif. and has a commercial manufacturing facility in Ohio. More information about Amylin Pharmaceuticals is available at www.amylin.com.

This press release contains forward-looking statements about Amylin, which involve risks and uncertainties. Our actual results could differ materially from those discussed herein due to a number of risks and uncertainties, including risks that metreleptin as a potential treatment option for diabetes and/or hypertriglyceridemia in pediatric and adult patients with rare forms of lipodystrophy will not be approved by the FDA; risks that we will not receive Priority Review of the BLA mentioned in this press release; risks that if Priority Review is granted it will not reduce the amount of time it takes the FDA to review the BLA; risks that our clinical trials will not be completed when planned, may not replicate previous results, may not be predictive of real world use or may not achieve desired end-points; risks that our preclinical studies may not be predictive; risks that our NDAs for product candidates or sNDAs for label expansion requests may not be submitted timely or receive FDA approval; and other risks inherent in the drug development and commercialization process. These and additional risks and uncertainties are described more fully in the Company's recently filed Form 10-K. Amylin disclaims any obligation to update these forward-looking statements.

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SOURCE Amylin Pharmaceuticals, Inc.