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    Drugs in Perspective: Myalept (metreleptin)


    Lipodystrophy is an orphan disease. Only an estimated few thousand patients have this disease worldwide, although robust epidemiologic data are not available.1,2 Lipodystrophy is a group of chronic, life-threatening disorders marked by abnormalities in fat tissue distribution and insufficient quantities of fat required for normal metabolic function.3 The 2 main types of lipodystrophy are generalized and partial lipodystrophy, which may be further categorized into congenital and acquired subtypes. Patients with congenital forms are born with little to no fat tissue, whereas acquired subtypes lose fat tissue over time. The loss of adipose tissue results in a deficiency in the hormone leptin, an adipocyte-derived hormone.5 A low level of leptin signals starvation and instructs the body to adapt, resulting in metabolic abnormalities and increased appetite, known as hyperphagia. Replacement of leptin does not correct the underlying fat deficiency.6 Severe lipodystrophy is associated with metabolic abnormalities including insulin resistance, hypertriglyceridemia, and hyperlipidemia, and can result in life-threatening comorbidities such as pancreatitis, progressive liver disease (ie, hepatic steatosis or cirrhosis), and/or accelerated cardiovascular diseases.5,8 Often in severe disease, traditional medications are less effective at targeting metabolic markers and do not control the average level of glycated hemoglobin (HbA1c) and lipid levels long term.1

    Read more Drugs in Perspective

    Myalept (metreleptin) is a leptin analogue with the same physiologic effects as leptin.11 Metreleptin was granted a priority review and was FDA approved on February 25, 2014, as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired lipodystrophy.7,10 Metreleptin is not approved for other forms of lipodystrophy, such as partial lipodystrophy or human immunodeficiency virus (HIV)-related lipodystrophy. The safety and effectiveness of metreleptin have not been established for the treatment of liver disease (including nonalcoholic steatohepatitis [NASH]), or for use in patients with metabolic disease without concurrent evidence of generalized lipodystrophy.

    The drug is available as a vial containing 11.3 mg metreleptin to deliver 5 mg per mL metreleptin when reconstituted.11 Metreleptin is only available through the Risk Evaluation and Mitigation Strategy (REMS) program for this drug due to the risk of developing neutralizing antibodies and lymphoma. The REMS program requires certification of prescribers and pharmacies and a prescription authorization form for each new prescription. Metreleptin should be administered once daily at the same time every day. Dosing for metreleptin is outlined in Table 1, and adjustments are based on clinical response.


    Table 1  Metreleptin recommended dosage

    Baseline weight

    Injection volume


    daily dose

    Dose adjustments


    daily dose

    Males and females <40 kg

    0.06 mg/kg

    (0.012 mL/kg)

    0.02 mg/kg

    (0.004 mL/kg)

    0.13 mg/kg

    (0.026 mL/kg)

    Males >40 kg

    2.5 mg

    (0.5 mL)

    1.25 mg (0.25 mL) to 2.5 mg (0.5 mL)

    10 mg

    (2 mL)

    Females >40 kg

    5 mg

    (1 mL)

    1.25 mg (0.25 mL) to 2.5 mg (0.5 mL)

    10 mg

    (2 mL)

    FormularyWatch/Source Ref 11



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