Metreleptin

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Metreleptin
Names
Trade namesMyalept, Myalepta, others
Other namesN-Methionylleptin; r-metHuLeptin, Mettreleptin (genetical recombination) (JAN JP)
Clinical data
Drug classLeptin analog[1]
Main usesLipodystrophy[1]
Side effectsLow blood sugar, weight loss[2]
Pregnancy
category
  • US: C (Risk not ruled out)[3]
Routes of
use
Subcutaneous injection
External links
AHFS/Drugs.comMonograph
US NLMMetreleptin
Legal
License data
Legal status
  • UK: POM (Prescription only) [4]
  • US: ℞-only [5]
  • EU: Rx-only [2]
  • In general: ℞ (Prescription only)
Chemical and physical data
FormulaC714H1167N19O221S6
Molar mass13746.46 g·mol−1

Metreleptin, sold under the brand name Myalept among others, is a medication used to treat lipodystrophy.[1] It is given by injection under the skin.[1] It is used together with diet.[2]

Common side effects include low blood sugar and weight loss.[2] Other side effects may include headache, abdominal pain, tiredness, and fever.[1] Severe side effects may include anaphylaxis and lymphoma.[1] It is a recombination form of leptin.[1]

Metreleptin was approved for medical use in the United States in 2014 and Europe in 2018.[1][2] In the United States it costs about 166,000 USD for a month of 11.3 mg as of 2021.[6] This amount in the United Kingdom costs the NHS about £70,000.[7]

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.[2]

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.[5]

Dosage

It is given at an initial dose of 2.5 mg in males over 40 kg and 5 mg in females over 40 kg.[1]

History

It has been approved in Japan.[8]

Research

Metreleptin is currently[when?] 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.[9] The FDA approved Metreleptin injection for treating complications of leptin deficiency in February 2014.[medical citation needed]

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).[10] 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.[11] 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.[12]

References

  1. 1.0 1.1 1.2 1.3 1.4 1.5 1.6 1.7 1.8 "Metreleptin Monograph for Professionals". Drugs.com. Retrieved 18 November 2021.
  2. 2.0 2.1 2.2 2.3 2.4 2.5 "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.
  3. "Metreleptin (Myalept) Use During Pregnancy". Drugs.com. 4 May 2020. Retrieved 28 May 2020.
  4. "Myalepta 3 mg powder for solution for injection - Summary of Product Characteristics (SmPC)". (emc). Retrieved 6 October 2020.
  5. 5.0 5.1 "Myalept- metreleptin injection, powder, lyophilized, for solution". DailyMed. 26 May 2020. Retrieved 6 October 2020.
  6. "Myalept Prices, Coupons & Patient Assistance Programs". Drugs.com. Retrieved 18 November 2021.
  7. "Metreleptin". SPS - Specialist Pharmacy Service. 3 February 2016. Retrieved 18 November 2021.
  8. 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.
  9. "Amylin Seeks FDA Approval for Metreleptin". diabetesincontrol.com. 11 April 2012.
  10. "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.[permanent dead link]
  11. 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.
  12. "NHS England » Metreleptin for congenital leptin deficiency (all ages)". www.england.nhs.uk. Retrieved 2019-01-18.

External links

External sites:
Identifiers: