Metreleptin for injection to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy

Abstract

The lipodystrophies represent a class of diseases characterized by leptin deficiency. Leptin deficiency is associated with a severe form of the metabolic syndrome characterized by dyslipidemia, insulin resistance, diabetes, and ovarian dysfunction. Metreleptin is the pharmaceutical derived product that has been approved by the Food and Drug Administration (FDA) to treat the severe metabolic abnormalities of the generalized forms of lipodystrophy. Herein we describe the properties of metreleptin, its use in patients, which includes the administration of the drug and how it may be acquired by medical professionals as well as its safety, tolerability, and properties. Finally, we speculate on future uses and development of metreleptin.

Keywords:

• lipodystrophy
• leptin
• metreleptin
• metabolic syndrome
• insulin resistance

Acknowledgements

We are grateful to the nurses and clinical fellows who have cared for our patients. We are also grateful to the following pharmaceutical companies who have made metreleptin available to us: Amgen, Bristol Myers Squib, Astra Zeneca and Aegerion.