Myalepta is a medicine used in addition to diet to treat lipodystrophy, where patients have loss of fatty tissue under the skin and build-up of fat elsewhere in the body such as in the liver and muscles. The medicine is used in: - adults and children above the age of 2 years with generalised (throughout the body) lipodystrophy (Berardinelli-Seip syndrome and Lawrence syndrome); - adults and children above the age of 12 years with partial (localised) lipodystrophy (including Barraquer-Simons syndrome), when standard treatments have failed. Myalepta contains the active substance metreleptin. Because the number of patients with the various forms of lipodystrophy is low, the diseases are considered ‘rare’, and Myalepta was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 17 July 2012. Further information on the orphan designations can be found on the European Medicines Agency’s website: ema.europa.eu/Find medicine/Human medicines/Rare disease designation ( [Bernardinelli-Seip syndrome](/en/medicines/human/orphan-designations/eu3121025): 17/07/12; [Lawrence syndrome](/en/medicines/human/orphan-designations/eu3121024): 17/07/12; [familial partial lipodystrophy](/en/medicines/human/orphan-designations/eu3121022): 17/07/12; [Barraquer-Simons syndrome](/en/medicines/human/orphan-designations/eu3121023): 17/07/12).
Therapeutic Indication
### Therapeutic indication Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: - with confirmed congenital generalised LD (Berardinelli-Seip syndrome) or acquired generalised LD (Lawrence syndrome) in adults and children 2 years of age and above - with confirmed familial partial LD or acquired partial LD (Barraquer-Simons syndrome), in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control.
Therapeutic Area (MeSH)
ATC Code
A16AA
ATC Item
氨基酸及其衍生物
Pharmacotherapeutic Group
Other alimentary tract and metabolism products
Active Substance (Summary)
INN / Common Names
| Substance | CAS | Monograph |
|---|---|---|
| Metreleptin | N/A | Metreleptin |
EMA Name
Myalepta
Medicine Name
Myalepta
Aliases
N/ANo risk management plan link.