Metreleptin

Metreleptin is an analog of the human hormone leptin, a neurohormone secreted by fat cells that plays a fundamental role in the regulation of energy homeostasis, fat and glucose metabolism and body weight. Metreleptin (recominbinant methionyl human leptin) therapy can have profound effects on improving insulin sensitivity, high trigylcerides, hyperglycemia and liver fat in patients with lipodystrophy who are not responsive to conventional lipid and glucose-lowering agents.

Amylin recently submitted the clinical and nonclinical sections of a rolling Biologics License Application (BLA) for metreleptin to treat diabetes and/or hypertriglyceridemia (high levels of triglycerides in the bloodstream) in patients with rare inherited or acquired forms of lipodystrophy.

The current status of our regulatory filing is indicated in the press release below:
Amylin Submits Clinical and Nonclinical Sections of Rolling Biologics License Application for Metreleptin to Treat Rare Forms of Lipodystrophy (December 20, 2010)

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.

About 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 that is normally secreted from fat cells that plays a key role in regulating metabolism.

Current treatment options for patients with lipodystrophy can be quite limited, with no current FDA-approved medication indicated for the treatment of lipodystrophy. Treatments today target the metabolic symptoms that lipodystrophy patients experience, and include a combination of dietary modification, anti-diabetic medications and lipid-lowering agents. However, these currently available treatments are often rendered marginally effective due to the severity of the condition. There is a significant unmet medical need for a therapy that effectively improves the metabolic abnormalities of these patients.

Expanded Access Program

Consistent with the severity and rare nature of this disorder, Amylin has received both orphan drug designation from FDA’s Office of Orphan Products Development, as well as fast track designation for use of metreleptin in patients with lipodystrophy.

Because metreleptin is not available for routine clinical use, and because of the high unmet medical need of these patients, we have worked to make this investigational medication available now under an expanded access pathway, an FDA-authorized treatment IND protocol. The treatment IND mechanism allows for access to investigational medications in special cases of unmet medical need. For more information on this program, please click here to Contact Us.