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Amylin Pharmaceuticals recently announced that it is participating in the fifth annual international Rare Disease Day to demonstrate the Company’s support for patients and families who are impacted by rare diseases. Celebrated around the world today, Rare Disease Day aims to raise awareness of the more than 7,000 known rare diseases and the effects of these diseases on patients’ lives, and to provide a platform of unity for rare disease patient communities.
In the U.S., a disease is considered rare, or orphan, if it affects fewer than 200,000 individuals. According to the National Institutes of Health Office of Rare Diseases Research, an estimated 25 to 30 million people in the U.S. have a rare disease. Most rare diseases have limited or no associated treatment options ‚Äď and, to date, there are no known cures for these diseases.
“We are honored to participate in activities to draw heightened attention to rare diseases and the critical need to drive research to better understand these diseases and develop effective therapies for patients,” said Daniel M. Bradbury, president and chief executive officer of Amylin Pharmaceuticals.
“At Amylin, we are proud to be advancing the development of a potential therapy for rare forms of lipodystrophy, an ‘ultra-orphan’ disease. We believe all patients ‚Äď whether they are impacted by one of the world’s most prevalent diseases, like type 2 diabetes, or one of the rarest ‚Äď deserve better treatment options.”
Amylin launches disease awareness website ‚Äď mylipodystrophy.com
To coincide with Rare Disease Day, Amylin also announced today the launch of¬†mylipodystrophy.com, a disease awareness website that is dedicated to rare forms of lipodystrophy, and serves as a resource for information about the disease for patients, caregivers and healthcare providers.
People with lipodystrophy lack fat tissue. This can be partial, affecting select areas of the body, or generalized, affecting the entire body. The physical appearance of people with lipodystrophy can therefore vary considerably. Fat tissue is a major endocrine organ producing important metabolic hormones such as leptin.
A lack of functioning fat tissue can lead to relative deficiency of leptin. Without¬†adequate¬†leptin function, the metabolic system, which regulates food intake, energy storage, and energy production, falls out of balance. Fat accumulates in the blood or organs, 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 to adequately treat the metabolic abnormalities that occur in lipodystrophy.¬†To help raise public awareness of rare diseases, Amylin is also featuring a Rare Disease Day series on its corporate blog, Building Blocks, available at¬†www.amylinbuildingblocks.com.
This series includes contributions from several experts in the rare disease field describing efforts underway to understand and combat rare diseases, as well as initiatives that are providing support for patient communities. Amylin is also participating in the¬†Global Genes Project’s¬†”Wear that you Care” denim campaign, encouraging its employees to wear jeans to work today to show support for the rare disease cause.
Amylin is developing metreleptin as a potential treatment option for diabetes and/or hypertriglyceridemia in pediatric and adult patients with rare forms of lipodystrophy. The Company plans to complete the rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) in the first half of this year.