Metreleptin with brand name Myalept is an anti-obesity intravenous injection which was approved by the US-FDA in Feb 2014 for the treatment of congenital and acquired generalized lipodystrophy. It is a synthetic analog of Leptin, an adipocyte hormone produced by fat cells and plays an important role in regulating energy homeostasis. This drug is recommended as a replacement therapy to treat the complications that arise from Leptin deficiency.
Lipodystrophy is a group of very rare syndromes characterized by a loss of fat tissue. In some patients, it is genetic which is known as congenital lipodystrophy and in others it may be acquired from different pathophysiological conditions, and in some cases due to unknown reasons. Generalized lipodystrophy is an orphan disease, also commonly referred to as abnormality of adipose tissue distribution, with a lack of fat tissue. It is characterized by a widespread loss of fat tissue under the skin. It can also be referred to as dyslipidemia, and is defined as abnormal amounts of lipids in blood. Most common form of dyslipidemia is hyperlipidemia, characterized by the elevated levels of lipids/fats in blood other than the adipose tissue. Due to this, there is a loss of fat tissue under the skin and this further leads to a deficit in the hormone leptin thereby causing multiple metabolic complications including significant decrease in blood glucose and triglyceride levels.
Leptin is a hormone which is synthesized by fat cells. This hormone regulates the amount of stored fat in the body. It exerts its effect by adjusting the sensation of hunger on one hand and the energy expenditures on the other hand. When the amount of fat stored reaches a certain level hunger is inhibited (satiety). Leptin is then secreted and circulates through the body which in turn activate leptin receptors in the arcuate nucleus of the hypothalamus. In human body energy expenditure is increased both by the signal to the brain, and directly via leptin receptors on peripheral targets. This signaling pathway decrease food intake and increase energy expenditure which terminates deposition of the fat into the Adipocytes.
Majority of the cases of lipodystrophy do not require any surgical intervention, as it can be treated with replacement therapy of the leptin analog. Myalept is one such analog of leptin made by recombinant DNA technology. This drug was evaluated in an open-label, single-arm study which included 48 patients with congenital or acquired generalized lipodystrophy and was proven to be safe and efficacious. The participants also had diabetes mellitus, hypertriglyceridemia, and/or elevated levels of fasting insulin. Myalept was administered as a subcutaneous injection once daily after reconstitution with Bacteriostatic Water for Injection (BWFI) in the patients. The trial showed reductions in HbA1c (a measure of blood sugar control) from 9.4% at baseline to 7.0% at end of study), fasting glucose, and triglycerides (from 500 mg/dl at baseline to 200mg/dl at end of study).
Some of the side effects manifested with usage of this drug included headache, weight loss, abdominal pain etc. Myalept is not approved for use in patients with HIV-related lipodystrophy or for patients with metabolic diseases, including diabetes mellitus and hypertriglyceridemia, without concurrent evidence of generalized lipodystrophy.
Due to the risks associated with the development of neutralizing antibodies and lymphoma, Myalept is available only through the Myalept Risk Evaluation and Mitigation Strategy (REMS) Program. Under this REMS program, prescribers must be certified with the program by enrolling in and completing training. Pharmacies must be certified with the program and only dispense Myalept after receipt of the Myalept REMS prescription Authorization form for each new prescription. This drug should not be used for patients who have general obesity that is not caused by a congenital leptin deficiency are allergic to Metreleptin or any of the ingredients in Myalept. Patients should contact their physician immediately if they develop any symptoms of an allergic reaction including a rash or itch (hives), or have symptoms like swelling of face, lips, tongue, or throat etc. Myalept is also approved with a medication guide and instructions for use that provides patients with important information about the medication. The guide would be distributed each time a patient gets a prescription.
Thus, considering the above mentioned advantages of the new drug Myalept, it has become the drug of choice today for correcting the leptin deficiency in patients with congenital generalized or acquired generalized lipodystrophy. Even though only 1 in 10 million people worldwide are reported to be suffering from this condition, this new treatment choice approved for the first time by the US FDA brings significant hope and adds a new dimension in the treatment of this disease.
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