European Commission Greenlights MYALEPTA for Lipodystrophy

According to a report by EuroInvestor, biopharmaceutical company Novelion Therapeutics, Inc. was recently granted permission to market their drug MYALEPTA. Novelion intends MYALEPTA to function as a method of treating leptin deficiency in certain cases of lipodystrophy. The drug functions as an “adjunct to diet,” a form of replacement therapy. MYALEPTA is the first of its kind in Europe where it has been approved. Keep reading to learn more, or follow the original story here for additional details.

What is Congenital Generalized Lipodystrophy?

Congenital generalized lipodystrophy (CGL) is a rare autosomal recessive condition of the skin. The most notable characteristic of the condition is that it greatly reduces the amount of body fat, emphasizing an extreme amount of muscularity. The amount of fat loss is often an indicator of how severe the condition is. Later in progression, CGL may cause fat to build up in the muscles and liver. Heart problems, liver disease, and insulin resistance may occur as a result. Other symptoms are dependent on the exact nature of a person’s CGL condition.

Current treatments for CGL focus on the management of symptoms. Dieting is the most common form of management. People living with CGL are recommended to maintain diets high in carbohydrates and low in fats. Corrective surgery may be used in some cases when patients desire or need to alter their appearance. Liver transplants may also become necessary at later stages.

Click here to learn more about congenital generalized lipodystrophy.


The recent approval for MYALEPTA comes after it had already been granted Orphan Drug designation in 2012. Now with the European Commission’s marketing authorization, MYALEPTA becomes the first (as well as the only) licensed treatment of its kind.

MYALEPTA is a “recombinant analog of human leptin.” It is a replacement therapy option for the normal diet recommended for CGL patients. It also boats the ability to treat leptin deficiency in patients with acquired generalized leptin deficiency, and familial partial lipodystrophy.

The drug therapy also received positive comments from the Committee for Medicinal Products for Human Use in May of last year. The US Food and Drug Administration has approved it for leptin deficiency but not yet for partial lipodystrophy.

A Treatment Milestone

An open-label, single-arm study evaluated MYALPETA in the treatment of 48 patients. The clinical data from this study formed the basis of the European Commission’s authorization. Further studies and a few safety measures will be required as conditions of the approval, but MYALEPTA appears set to move forward.

Meanwhile, experts on lipodystrophy describe the new treatment as a major step forward for patients. Lipodystrophy typically requires a complex treatment plan. Typically this involves life style changes on the part of the patient. Strict dieting is usually required. Now, however, doctors will be able to offer patients an option which reduces the need for lifestyle changes.

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