Metreleptin Treatment in Patients with Non-HIV Associated Lipodystrophy

Lipodystrophies are a heterogeneous group of disorders characterized by congenital or acquired loss of adipose tissue. Recently, metreleptin, a recombinant human leptin analog, has been approved for the treatment of patients with generalized lipodystrophy. Leptin is an adipokine which has a fundamental role in glucose and lipid homeostasis. Metreleptin treatment has been demonstrated to improve metabolic abnormalities such as hyperglycemia, hypertriglyceridemia, increased hepatic fat content and elevated liver enzymes alanine transaminase and aspartate transaminase in patients with generalized lipodystrophy, and to correct hyperphagia that likely occurs as a result of leptin deficiency. Limited data has also suggested that metreleptin treatment might be beneficial on metabolic abnormalities in patients with partial lipodystrophy. This review focuses on potential benefits of metreleptin in various forms of non-HIV associated lipodystrophy. Safety issues have been discussed. Recent patent submissions have also been reviewed.