LIPODYSTROPHY ORPHAN DRUG PROGRAM
Patients with severe lipodystrophy, a very rare metabolic condition, have abnormalities in adipose tissue (fat) distribution with loss of subcutaneous fat. They have multiple metabolic disorders – extreme insulin resistance, very high triglyceride levels, diabetes and steatosis (fat accumulation in tissues like liver and muscle) – that are associated with increased risk of severe pancreatitis, early diabetes complications, cirrhosis and early cardiovascular death. Because these patients have too little fat, rather than too much, they have a deficiency of leptin, a hormone that is normally secreted from fat.
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, antidiabetic medications and lipid-lowering agents. However, these currently available treatments are often not effective for these patients because of the severity of their disease. There is a significant unmet clinical need for a therapy that effectively improves the metabolic disorders of these patients.
Compelling clinical data has demonstrated that replacing leptin in patients with lipodystrophy can have profound effects to improve insulin sensitivity, high triglycerides, hyperglycemia and liver fat in patients otherwise not responsive to conventional lipid and glucose lowering agents. Because of the rarity of this condition, Amylin has received an orphan drug designation from FDA’s Office of Orphan Products Development for use of metreleptin, a leptin analogue, 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, contact Jean Chan via e-mail Jean.Chan@amylin.com or phone (858) 754-4407.