EMA Panel Recommends Metreleptin for Leptin Deficiency

Troy Brown, RN

June 01, 2018

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended marketing authorization for metreleptin (Myalepta, Aegerion Pharmaceuticals BV) for the treatment of complicaitons of leptin deficiency in those with generalized or partial lipodystrophy. Patients with the disorder can develop severe metabolic abnormalities, including hypertriglyceridemia, insulin resistance, and/or diabetes.

Metreleptin, which was granted orphan medicine status on July 17, 2012, is a recombinant human leptin analogue (ATC code: A16AA07) that decreases different types of fat in the body and reduces their accumulation in tissues, including liver and muscle, by binding to and activating the human leptin receptor. It will be distributed as an 11.3-mg powder for injectable solution.

Metreleptin lowers glycated hemoglobin and triglyceride levels in those with lipodystrophy. The most frequently seen adverse effects are hypoglycemia, weight loss, injection site reactions, and the formation of neutralizing antibodies.

The US Food and Drug Administration approved metreleptin for generalized lipodystrophy on February 25, 2014.

The complete indication for metreleptin is as an adjunct to diet as a replacement therapy to treat complications of leptin deficiency in patients with lipodystrophy (LD) with confirmed congenital generalized LD (Berardinelli-Seip syndrome) or acquired generalized LD (Lawrence syndrome) in adults and in children aged 2 years and older or in adults and children aged 12 years and older with confirmed familial partial LD or acquired partial LD (Barraquer-Simons syndrome) for whom standard treatments have failed to establish adequate metabolic control.

The CHMP proposes that metreleptin be prescribed by physicians with experience in the diagnosis and management of metabolic disorders.

The summary of product characteristics will include detailed recommendations for use of the product and will be published in the European public assessment report, which will be made available in all official European Union languages after the European Commission grants marketing authorization.

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