Metreleptin in lipodystrophy: a profile of its use
Metreleptin [Myalepta® (EU); Myalept® (USA)] is a recombinant analogue of human leptin and currently the only drug available for the specific treatment of lipodystrophy (LD). In the EU, metreleptin (administered once daily via subcutaneous injection) is indicated as replacement therapy to treat the complications of leptin deficiency in patients aged ≥ 2 years with generalized LD and in patients aged ≥ 12 years with partial LD who have failed to achieve adequate metabolic control with standard treatments. Its use in these rare settings is supported by data from open-label clinical studies and clinical practice, with the totality of evidence indicating that metreleptin improves metabolic abnormalities associated with generalized or partial LD, including in paediatric patients. Other potential benefits include improved hepatic parameters/disease, nephropathy and survival, although the impact of the drug on these outcomes would benefit from further analysis. Metreleptin is generally well tolerated.
The manuscript was reviewed by: L. Roever, Department of Clinical Research, Federal University of Uberlândia, Uberlândia, Brazil; F. Santini, Endocrinology Unit, Obesity and Lipodystrophy Center, University Hospital of Pisa, Pisa, Italy. During the peer review process, Aegerion Pharmaceuticals B.V., the marketing-authorization holder of metreleptin, was also offered an opportunity to provide a scientific accuracy review of their data. Changes resulting from comments received were made on the basis of scientific and editorial merit.
Compliance with ethical standards
The preparation of this review was not supported by any external funding.
Conflict of interest
E. D. Deeks is an employee of Adis/Springer, is responsible for the article content and declares no conflicts of interest.
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