Metreleptin

Metreleptin, sold under the brand name Myalept among others, is a synthetic analog of the hormone leptin used to treat various forms of dyslipidemia. It has been approved in Japan for metabolic disorders including lipodystrophy and in the United States as replacement therapy to treat the complications of leptin deficiency, in addition to diet, in patients with congenital generalized or acquired generalized lipodystrophy.[4]

Metreleptin
Clinical data
Trade namesMyalept, Myalepta
Other namesN-Methionylleptin; r-metHuLeptin, Mettreleptin (genetical recombination) (JAN JP)
AHFS/Drugs.comMonograph
License data
Routes of
administration		Subcutaneous injection
ATC code
Legal status
Legal status
  • UK: POM (Prescription only) [1]
  • US: ℞-only [2]
  • EU: Rx-only [3]
  • In general: ℞ (Prescription only)
Identifiers
CAS Number
DrugBank
ChemSpider
  • none
UNII
KEGG
ChEMBL
Chemical and physical data
FormulaC714H1167N19O221S6
Molar mass13746.46 g·mol−1

The most common side effects include hypoglycaemia (low blood glucose) and weight loss.[3]

Medical uses

In the European Union, metreleptin is indicated in addition to diet to treat lipodystrophy, where people have a loss of fatty tissue under the skin and a build-up of fat elsewhere in the body such as in the liver and muscles. It is used in adults and children above the age of two years with generalised lipodystrophy (Berardinelli-Seip syndrome and Lawrence syndrome); and in adults and children above the age of twelve years with partial lipodystrophy (including Barraquer-Simons syndrome), when standard treatments have failed.[3]

In the United States, it is indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in people with congenital or acquired generalized lipodystrophy.[2]

Research

Metreleptin is currently being investigated for the treatment of diabetes and/or hypertriglyceridemia, in patients with rare forms of lipodystrophy, syndromes characterized by abnormalities in adipose tissue distribution, and severe metabolic abnormalities.[5] The FDA approved Metreleptin injection for treating complications of leptin deficiency in February 2014.

In a three-year study of metreleptin in patients with lipodystrophy organized by the National Institute of Diabetes and Digestive and Kidney Diseases at the National Institutes of Health, metreleptin treatment was associated with a significant decrease in blood glucose (A1c decreased from 9.4% at baseline to 7.0% at study end) and triglyceride concentration (from 500 mg/dl at baseline to 200 mg/dl at study end).[6] Metreleptin is effective in most patients with generalized lipodystrophy where circulating leptin levels are extremely low. Analogous to insulin replacement for patients with type 1 Diabetes, metreleptin restores the function of a deficient hormone. However, in patients with partial lipodystrophy where there is only a relative leptin deficiency, the response to metreleptin is not universal.[7] This may or may not be due to anti-leptin antibodies.

NHS England will commission metreleptin treatment for patients (all ages) with congenital leptin deficiency from April 1, 2019.[8]

References
  1. "Myalepta 3 mg powder for solution for injection - Summary of Product Characteristics (SmPC)". (emc). Retrieved 6 October 2020.
  2. "Myalept- metreleptin injection, powder, lyophilized, for solution". DailyMed. 26 May 2020. Retrieved 6 October 2020.
  3. "Myalepta EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 6 October 2020. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  4. Chou K, Perry CM (June 2013). "Metreleptin: first global approval". Drugs. 73 (9): 989–97. doi:10.1007/s40265-013-0074-7. PMID 23740412. S2CID 7740045.
  5. "Amylin Seeks FDA Approval for Metreleptin". diabetesincontrol.com. 11 April 2012.
  6. "Amylin to Present Data Showing Investigational Metreleptin Treatment Led to Long-Term Improvements in Diabetes and Lipid Control in Patients with Lipodystrophy". Press Release. Amylin Pharmaceuticals. 2011-04-15. Retrieved 2011-10-27.
  7. Meral, Rasimcan; Malandrino, Noemi; Walter, Mary; Neidert, Adam H; Muniyappa, Ranganath; Oral, Elif Arioglu; Brown, Rebecca J (2021-10-22). "Endogenous Leptin Concentrations Poorly Predict Metreleptin Response in Patients with Partial Lipodystrophy". The Journal of Clinical Endocrinology & Metabolism. doi:10.1210/clinem/dgab760. ISSN 0021-972X.
  8. "NHS England » Metreleptin for congenital leptin deficiency (all ages)". www.england.nhs.uk. Retrieved 2019-01-18.
  • "Metreleptin". Drug Information Portal. U.S. National Library of Medicine.
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