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.