Myalepta (Metreleptin): New Hope For Patients With Lipodystrophy
Myalepta (generic: metreleptin), the first approved leptin replacement therapy, is offering new hope for these patients.
Lipodystrophy is a rare metabolic disorder in which patients suffer from severe metabolic disturbances due to leptin deficiency.
Global Approvals And Applications
Myalepta was initially approved by the FDA in the United States in 2014 for the treatment of leptin deficiency complications in patients with congenital or acquired generalized lipodystrophy.
Subsequently, in 2018, the European Medicines Agency (EMA) also approved a marketing application for the drug, expanding its global accessibility.
In February 2024, Health Canada (Health Canada) approved the use of Myalepta, further broadening its application.
Mechanism Of Action And Indications For Myalepta
Myalepta metreleptin is a recombinant human leptin analog that ameliorates severe metabolic disorders by supplementing patients with deficiencies of leptin, a key hormone that regulates appetite, metabolism, and energy balance. Its approved indications include:
Congenital leptin deficiency (CLD): a rare genetic disorder caused by mutations in the LEP gene in which patients present with extreme obesity, insulin resistance, and hypogonadism.
Acquired generalized lipodystrophy (AGL): a disease characterized by generalized adipose tissue loss, severe insulin resistance and fatty liver, often accompanied by hypertriglyceridemia and diabetes.
Safety And Precautions
In clinical trials, Metreleptin has shown promising efficacy, but some adverse effects, including weight loss, hypoglycemia, and fatigue, have been observed.
The FDA black box warning suggests that it may pose a risk of T-cell lymphomas (especially in patients with AGL) and that immune status needs to be assessed and monitored regularly prior to treatment.
Therefore, patients should be guided by a healthcare professional when using this drug to ensure safety and efficacy.
ACCESSIBILITY AND FUTURE OUTLOOK
Treatment options for patients with lipodystrophy have improved significantly with the worldwide approval and adoption of Myalepta, which is manufactured by Amryt Pharma and is currently approved in multiple countries in Europe and the United States.
Although annual treatment costs run into the hundreds of thousands of dollars, health insurance coverage and patient assistance programs are expected to ease the burden.
However, further studies are needed to evaluate its long-term efficacy and safety, as well as to explore additional possible indications.
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Our efforts to improve the affordability of Metreleptin drug aim to ensure that more patients can benefit from this important medication.
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